Hope for a precision medicine for ADNP syndrome 23 September 2026

Hope for a precision medicine for ADNP syndrome 23 September 2026 https://rarerevolutionmagazine.com/hope-for-a-precision-medicine-for-adnp-syndrome/ Genie and Rowland’s rare disease journey Typical of many busy families today, Genie Egerton-Warburton posts notes and calendars to a crowded household bulletin board. But some of the items on the Warburton board are unusual. There are sessions with occupational, educational, and physical therapists, visiting nurse and doctor appointments and a variety of reminders and charts. These entries are for Rowland, her 14-year-old son with a rare neurological disorder

The shift in antibody drug discovery From affinity to function, antibody discovery has shifted toward programmable biologics engineered to survive human physiology and execute complex therapeutic tasks at scale. Written byAndrea Corona

https://www.drugdiscoverynews.com/the-shift-in-antibody-drug-discovery-17021?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-83AGvtXKtrzP-Raxll3xEATtKmqRAOxHlAFxFypDaoeJJpL-eZLJWugQ9s9dWYtOBjvwbyx0BYyKQqs12z7E0Y3q-xSw&_hsmi=441645340&utm_content=441645340&utm_source=hs_email For three decades, the primary goal of antibody discovery was the refinement of binding affinity, maximizing the strength with which a monoclonal antibody (mAb) locked onto its target. But as we move through 2026, clinical milestones indicate a definitive pivot toward functional engineering. The modern antibody is no longer just a simple antagonist; it is a programmable scaffold designed to help overcome the physiological hurdles that once rendered most biologics ineffective.

Antibody discovery moves toward de novo design From computationally designed repertoires to AI-driven specificity prediction and multispecific therapeutics, antibody researchers are developing new ways to design molecules for the biology they need to influence. Written byBree Foster, PhD and Andrea Corona

Antibody discovery moves toward de novo design From computationally designed repertoires to AI-driven specificity prediction and multispecific therapeutics, antibody researchers are developing new ways to design molecules for the biology they need to influence. Written byBree Foster, PhD and Andrea Corona https://www.drugdiscoverynews.com/antibody-discovery-moves-toward-de-novo-design-17541?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-9Qcy62gGqVoCB1arOfC3hVcscePxYUMwVMX6d0jxbcxdo_MSZ8WTV3u8VGdR8BHdt2r3ID73ba_PLH99Fkxw766IBMug&_hsmi=441645340&utm_content=441645340&utm_source=hs_email What if researchers could design antibodies with specific biological functions rather than simply search for molecules that bind a target? That question ran through The Antibody Series 2026 (TAS 2026), where speakers explored how computational design, AI, and increasingly complex antibody formats could expand what these therapies can do. But the conference also underscored a persistent challenge. Mark van Dijk, Head of Discovery at Fairjourney Bio, told DDN that antibody technology may no longer be the main limitation. Instead, “the biology is the limitation.”

A great antibody is not necessarily a great drug Antibody discovery is becoming less about finding the strongest binder and more about finding molecules that can survive the journey to the clinic. Written byBree Foster, PhD

A great antibody is not necessarily a great drug Antibody discovery is becoming less about finding the strongest binder and more about finding molecules that can survive the journey to the clinic. Written byBree Foster, PhD For years, antibody discovery has largely revolved around one question: Can the molecule bind its target and produce the desired biological effect? Increasingly, researchers are asking another question much earlier in the process: Can that antibody actually become a drug? Maria Gonzalez-Pajuelo, cofounder and Executive Vice President of Technology and Scientific Office at FairJourney Bio, told DDN that developability has become a much more important consideration during antibody discovery, rather than an issue left until later-stage development. https://www.drugdiscoverynews.com/a-great-antibody-is-not-necessarily-a-great-drug-17548

Navigating GLP-1 Switches: A Guide for Primary Care

https://www.medscape.com/viewarticle/navigating-glp-1-switches-guide-primary-care-2026a100107z?ecd=WNL_trdalrt_pos1_ous_260930_etid8746389&uac=148436CN&impID=8746389 As GLP-1 receptor agonists and dual incretin agents become more common in treating obesity and type 2 diabetes, primary care providers are handling more medication switches. The reason may be insurance coverage, ongoing gastrointestinal side effects, inadequate response, or the need for better blood sugar control or weight support. Whatever the driver, switching is not simply trading one prescription for another. It requires planning around half-lives, dose history, side effects, and the risk for blood sugar swings.

Clinical Crossroads of IDH-Mutant Glioma Care: Navigating Real-World Challenges and Complexities CME Philadelphia Marriott Downtown Friday, November 13, 2026 | 7:00 am - 8:15 am ET

https://na.eventscloud.com/website/99069/ The management of IDH-mutant glioma is evolving rapidly from traditional toxic therapies toward targeted molecular treatments and earlier intervention. Let our distinguished faculty guide you on effective integration of novel targeted therapies into your practice and how to navigate complex clinical scenarios through enhanced multidisciplinary coordination. SNO has reviewed and approved this symposium as appropriate for presentation as an Independent Supported Session. The session constitutes the content and views of the sponsor and is not part of the official 2026 SNO Annual Meeting program.

Charting New Frontiers in Advanced Breast Cancer: Milestones, Missions, and Mastery Authors: Paolo Tarantino, MD, PhD; Kevin Kalinsky, MD, MS, FASCO; Sibylle Loibl, MD, PhD

https://www.medscape.org/viewarticle/charting-new-frontiers-advanced-breast-cancer-milestones-2026a1000fu7?page=1&src=mkmcmr_driv_stan_mscpedu_260930-OUS-HONC-charting-new-frontiers-advanced-breast-cancer-milestones-2026a1000fu7-cta&uac=148436CN

CDER-What’s New in Regulatory Science-Issue 1, 2026 (+++++) +...

5-FY26 Nonclinical Human Cardiac New Approach Methodologies (NAMs) Predict Vanoxerine-Induced Proarrhythmic Potential https://pubmed.ncbi.nlm.nih.gov/40863351/ Garcia MI, Bhardwaj B, Dame K, Charwat V, Siemons BA, Goswami I, Ismaiel OA, Mistry S, Feaster TK, Healy KE, Ribeiro AJS, Blinova K. J Cardiovasc Dev Dis. 2025 Jul 26;12(8):285. Cardiac New Approach Methodologies (NAMs) were evaluated for their ability to detect drug-induced cardiac arrhythmic events that were not identified in nonclinical animal models or Phase I-II clinical trials. More specifically, the NAM utilized human-induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) and electrophysiological measurements. The drug examined, vanoxerine, has known cardiac risks that were not detected until late-stage clinical trials. In this study, researchers demonstrated for the first time that this compound triggered proarrhythmic events in a human-relevant nonclinical model. These findings demonstrate that this nonclinical cardiac NAM can successfully recapitulate clinical outcomes associated with vanoxerine. 6-FY26 Nitrosamine Ames Data Review and Method Development: Proceedings of a US FDA/HESI Workshop https://pubmed.ncbi.nlm.nih.gov/41689482/ Atrakchi, Aisar; Puglisi, Raechel; Bercu, Joel; Cheung, Jennifer; Czich, Andreas; Froestchl, Roland; Davis-Bruno, Karen; Heflich, Robert H; Kobets, Tetyana; McGovern, Timothy J; Lynch, Anthony; Selby, Max; Schuler, Maik; Silveira, Gabriela De Oliveira; Vespa, Alisa; Whomsley, Rhys; Chen, Connie L. Mutagenesis. 2026 Jun 27;41(4):225-236. Due to concerns over the sensitivity of the standard Ames test (OECD Test Guideline 471) in detecting the mutagenic and carcinogenic potential of N-nitrosamines (NAs) — including NA Drug Substance-Related Impurities (NDSRIs) found in marketed pharmaceuticals — the FDA's Center for Drug Evaluation and Research (CDER) Office of New Drugs (OND) and the Health and Environmental Sciences Institute's Genetic Toxicology Technical Committee (HESI/GTTC) co-organized a workshop to discuss optimized Ames test conditions for evaluating NAs and NDSRIs, with sessions addressing key parameters such as metabolic activation methods and tester strain selection. This report summarizes the key takeaways from that workshop. 7-FY26 Beyond QSARs: Quantitative Knowledge-Activity Relationships (QKARs) for enhanced drug toxicity prediction https://pubmed.ncbi.nlm.nih.gov/41025529/ Li, Ting; Qu, Yanyan; Chen, Alexander; Thakkar, Shraddha; Li, Dongying; Tong, Weida. Toxicol Sci. 2025 Dec 1;208(2):269–278. This study introduces Quantitative Knowledge-Activity Relationships (QKARs), a novel computational framework that predicts drug toxicity using domain-specific knowledge rather than relying solely on chemical structure as traditional Quantitative Structure-Activity Relationships (QSARs) do. QKAR models were developed for two key toxicity endpoints, drug-induced liver injury (DILI) and drug-induced cardiotoxicity (DICT). Leveraging the advances in AI, including text embedding and generative AI, were found to consistently outperform QSARs, particularly in distinguishing drugs with similar structures but different toxicity profiles. The study also explored integrating knowledge-based and structure-based representations (Q(K+S)ARs), demonstrating that QKARs offer a robust and promising alternative to QSARs for enhanced drug toxicity prediction and risk assessment. 8-FY26 Optimizing Oligonucleotide Therapeutics: A Model-Informed Drug Development Perspective https://pubmed.ncbi.nlm.nih.gov/42083118/ Yuan, Ye; Sharma, Vishnu; Bhattaram, Venkatesh Atul; Pan, Xiaolei; Earp, Justin; Wang, Yun; Liu, Jiang; Zhu, Hao. Clin Transl Sci. 2026 May;19(5):e70570 Oligonucleotide therapies, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers, present unique drug development challenges, particularly the disconnect between systemic exposure and tissue activity, where these therapeutics are rapidly cleared from circulation yet persist intracellularly with sustained effects, complicating dose optimization and efficacy prediction especially given limited clinical data from rare disease indications. This review examines how Model-Informed Drug Development (MIDD) has emerged as a critical solution to these challenges across FDA-approved oligonucleotide therapies, demonstrating how quantitative modeling has been successfully applied to bridge the PK-PD disconnect, inform endpoint selection for accelerated approvals, guide dosing strategies for general and special populations, and ultimately advance the clinical and regulatory success of this therapeutic class. 9-FY26 Using real-world data to predict findings of an ongoing phase IV trial: glycemic control of semaglutide versus standard of care https://pubmed.ncbi.nlm.nih.gov/41161770/ Kattinakere Sreedhara S, Schneeweiss S, D'Andrea E, Weberpals JG, DiCesare EC, Patorno E, Tsacogianis T, Bradley M, Concato J, Wang SV. BMJ Open Diabetes Res Care. 2025 Oct 29;13(5):e005180. Using national claims data (Optum Clinformatics, 2017–2022), this study emulated the design of the ongoing SEPRA trial to compare once-weekly injectable semaglutide against standard-of-care (SoC) medications for glycemic control (A1C <7%) in adults with type-2 diabetes on metformin monotherapy. Among 1,144 propensity score-matched pairs, semaglutide initiators were 30% more likely to achieve glycemic control (RR 1.30, 95% CI: 1.16–1.45) and showed a slightly greater A1C reduction (1.3% vs. 1.1%) compared to SoC, findings that were consistent with interim SEPRA trial results released after the study protocol was preregistered. These results support the value of well-designed, preregistered non-randomized studies using fit-for-purpose real-world data as effective complements to pragmatic randomized controlled trials. Impact Stories https://www.fda.gov/drugs/regulatory-science-action/impact-stories?utm_medium=email&utm_source=govdelivery

April - June 2026 | New Safety Information or Potential Signals of Serious Risks Identified by the FDA Adverse Event Monitoring System (AEMS) [Formerly FDA Adverse Event Reporting System (FAERS)]

https://www.fda.gov/drugs/fda-adverse-event-monitoring-system-aems/april-june-2026-new-safety-information-or-potential-signals-serious-risks-identified-fda-adverse

FDA Releases FY 2024 Pesticide Residue Monitoring Report (+... ...)

https://www.fda.gov/food/hfp-constituent-updates/fda-releases-fy-2024-pesticide-residue-monitoring-report?utm_medium=email&utm_source=govdelivery FDA Releases FY 2024 Pesticide Residue Monitoring Report September 30, 2026 Today, the FDA made available its annual Pesticide Residue Monitoring Program Report for Fiscal Year 2024, summarizing findings from FDA testing of human and animal foods for 781 different pesticides and selected industrial compounds from Oct. 1, 2023, through Sept. 30, 2024. Companies that grow, produce or manufacture food products sold in the U.S. must comply with applicable Environmental Protection Agency and FDA regulations. To protect public health, the FDA’s pesticide residue monitoring program tests FDA-regulated foods shipped in interstate commerce to determine whether they comply with pesticide tolerances, or maximum residue levels, set by EPA. If the FDA finds that the amount of pesticide residue detected on a food exceeds any existing tolerance, or has no established tolerance, the FDA will take action as appropriate. Monitoring focuses on raw agricultural foods of dietary importance (i.e., foods that comprise the greater part of the U.S. diet and can most contribute to pesticide exposure in people) and foods consumed in large amounts by infants and young children. Pesticide Residue Monitoring Report and Data for FY 2024 https://www.fda.gov/food/pesticides/pesticide-residue-monitoring-report-and-data-fy-2024?utm_medium=email&utm_source=govdelivery Setting Tolerances for Pesticide Residues in Foods https://www.epa.gov/pesticide-tolerances/setting-tolerances-pesticide-residues-foods?utm_medium=email&utm_source=govdelivery Pesticides are widely used in producing food. These pesticides may remain in small amounts (called residues) in or on fruits, vegetables, grains, and other foods. To ensure the safety of the food supply for human consumption, EPA regulates the amount of each pesticide that may remain in and on foods. This webpage briefly describes how EPA sets limits, called tolerances, for pesticide residues in foods and animal feeds. For FY 2024, findings show that the levels of pesticide chemical residues in the U.S. food supply are generally in compliance with EPA pesticide tolerances. Overall Findings Human Food Samples: 3,528 total samples (893 domestic food samples from 46 states and 2,635 import food samples from 76 countries/economies). 98.2% of domestic samples and 84.4% of import samples were compliant with federal regulations (below EPA tolerances). No pesticide chemical residues were detected in 36.8% of domestic samples and 36.5% of import samples. Historically, the violation rate for imported foods has been higher than for domestic foods, and the FY 2024 results continue that trend. This higher violation rate affirms the risk-based approach, which prioritizes for sampling, among other factors, imported food products that are more likely to contain violative pesticide chemical residues, and the countries more likely to export them. This may be based on past problem areas, findings from state and federal monitoring, and foreign pesticide usage data. Animal Food Samples: 306 total samples (153 domestic food samples from 24 states and 153 import samples from 13 countries). 93.5% of domestic samples and 91.5% of import samples were compliant with federal regulations (below EPA tolerances). No pesticide chemical residues were detected in 47.1% of domestic samples and 42.5% of import samples. Focused Sampling In FY 2024, the FDA conducted pesticide analyses for the “Domestically Produced Animal-Derived Foods” assignment. The FDA collected and analyzed 110 samples of selected animal-derived domestic foods, consisting of 29 milk, 34 shell egg, 19 honey and 28 game meat samples. No violative pesticide chemical residues were found in any of these foods, and 93.6% of the samples contained no pesticide residues. Pesticide Report Data Dashboard FY 2024 pesticide monitoring data are available to view on the FDA Pesticide Report Data Dashboard, a data visualization tool that enables users to interact with the tables and figures presented in the report and more directly view the data underlying the summaries. The FDA developed the dashboard in 2025 to support the agency’s commitment to transparency and enhancement of the food chemical safety program. The FDA plans to update the FDA Pesticide Report Data Dashboard next fiscal year to visualize multiple years of data. https://www.fda.gov/food/pesticides/fda-pesticide-report-data-dashboard?utm_medium=email&utm_source=govdelivery Pesticide Residue Monitoring Program Reports and Data https://www.fda.gov/food/pesticides/pesticide-residue-monitoring-program-reports-and-data?utm_medium=email&utm_source=govdelivery Pesticide Residue Monitoring Program Questions and Answers https://www.fda.gov/food/pesticides/pesticide-residue-monitoring-program-questions-and-answers?utm_medium=email&utm_source=govdelivery Pesticides https://www.fda.gov/food/chemical-contaminants-pesticides/pesticides?utm_medium=email&utm_source=govdelivery FDA Total Diet Study (TDS) https://www.fda.gov/food/reference-databases-and-monitoring-programs-food/fda-total-diet-study-tds?utm_medium=email&utm_source=govdelivery

No, organic eggs are not more nutritious or necessarily healthier than regular eggs. They are just far more expensive. MarthaStewart.com | September 29, 2026

https://geneticliteracyproject.org/2026/09/29/no-organic-eggs-are-not-more-nutritious-or-necessarily-healthier-than-regular-eggs-they-are-just-far-more-expensive/ Organic eggs cost considerably more than conventional ones, but that premium mostly pays for how the hens are raised, not for a more nutritious egg, as Martha Stewart’s Randi Gollin reports. When it comes to nutrition, what the hen eats matters far more than the word on the carton. The organic label means hens eat organic feed, made without grains grown with synthetic pesticides or fertilizers and without GMO grains. They’re also raised under USDA welfare rules, tightened starting in 2023 through the Organic Livestock and Poultry Standards, which require outdoor access, ban cages and prohibit forced molting. Bigger space requirements and pricier organic grain explain the higher price tag.

Viewpoint: 7-OH controversy: An opioid is available over-the-counter–even at gas stations. Should we be concerned? The Hill | September 29, 2026

https://geneticliteracyproject.org/2026/09/29/viewpoint-7-oh-controversy-an-opioid-is-available-over-the-counter-even-at-gas-stations-should-we-be-concerned/ A potent opioid is sitting on gas station shelves next to the beef jerky, and anyone with $20 can buy it, notes Liberty Vittert in an opinion piece for The Hill. To prove the point, Vittert bought a package of 7-OH, short for 7-hydroxymitragynine, in about 90 seconds. “The clerk rang it up between a coca cola and some beef jerky for my dog. He never even looked up,” she writes.

Pharmalittle: We’re reading about Novo licensing a Chinese obesity pill, Merck pulling an antibiotic, and more Roche stopped work on an obesity drug candidate after trial data fell short

https://www.statnews.com/pharmalot/2026/09/29/novo-licenses-obesity-pill-from-china-merck-pulls-antibiotic/ By Ed SilvermanSept. 29, 2026 Pharmalot Columnist, Senior Writer

Child health is the national security crisis we keep ignoring Chronic illness, anxiety, depression, and other health problems threaten our future safety

https://www.statnews.com/2026/09/29/childrens-health-national-security-military-labor-workforce-policy-fixes/ By Tina L. Cheng, James M. Perrin, and Stanley McChrystalSept. 29, 2026 Cheng is the B.K. Rachford professor and chair of pediatrics, University of Cincinnati, and director of the Cincinnati Children’s Research Foundation. Perrin is professor of pediatrics emeritus, Harvard Medical School, and the John C. Robinson distinguished chair in pediatrics, Mass General Brigham for Children. McChrystal is a former commander of U.S. and coalition forces in Afghanistan and founder of the McChrystal Group.

She’s spent two decades talking to parents who don’t vaccinate their children. Here’s what she’s learned A sociologist on why some families skip immunizations, and why ‘misinformation’ isn’t really to blame

She’s spent two decades talking to parents who don’t vaccinate their children. Here’s what she’s learned A sociologist on why some families skip immunizations, and why ‘misinformation’ isn’t really to blame By Nicholas FlorkoSept. 29, 2026 Trust-in-Science Reporter https://www.statnews.com/2026/09/29/understanding-vaccine-skepticism-in-parents/

UniQure’s gene therapy continues to slow Huntington’s progression after four years But the magnitude of the treatment’s benefit waned compared to a similar analysis from a year ago

UniQure’s gene therapy continues to slow Huntington’s progression after four years But the magnitude of the treatment’s benefit waned compared to a similar analysis from a year ago https://www.statnews.com/2026/09/29/uniqure-huntingtons-gene-therapy-slows-disease-progression-four-years/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-9u8GCVduAdt-avf7m3Kw1QbeXiCMqIrbuy2DSY82drp-okseb1VwLj82pHtTRXZd8R4Ihh7blfLBz8WQZZXWXAinQadA&_hsmi=441553206&utm_content=441553206&utm_source=hs_email By Adam FeuersteinSept. 29, 2026 Adam Feuerstein, a senior writer and biotech columnist, is the author of Adam’s Biotech Scorecard, a subscriber-only newsletter about the crossroads of drug development, business, Wall Street, and biotechnology. UniQure's Huntington's benefit is less pronounced four years in UniQure’s experimental Huntington’s gene therapy AMT-130 continued to slow disease progression four years after treatment. But the magnitude of the benefit declined from a year earlier — which could potentially complicate the therapy’s regulatory review, STAT’S Adam Feuerstein writes. Among 12 patients given a high dose, AMT-130 slowed progression by 44% compared with an external natural history control group, a difference that was not statistically significant. At three years, however, the company had reported a slowing of 75%, which was statistically significant. This decrease in efficacy can partly be attributed to faster-progressing patients in the historical control group dropping out of the study, UniQure CEO Matthew Kapusta said. Still, other measures were more encouraging: AMT-130 maintained a 61% slowing in loss of functional capacity, while levels of NfL, a marker of neuronal damage, stayed close to the baseline.

Accelerating equitable cancer genomics and precision oncology in health care and research: a Lancet Oncology Commission Raffaella Casolino, MD PhDa Send email to raffaella.casolino@glasgow.ac.uk ∙ Joaquin Mateo, MD PhDc ∙ Prof Elisabeth G E de Vries, MDd ∙ Amber Johns, BMedSci PhDa ∙ Melanie Courtot, PhDe,f,g ∙ Prof Rita T Lawlor, PhDj ∙ et al.

Accelerating equitable cancer genomics and precision oncology in health care and research: a Lancet Oncology Commission Raffaella Casolino, MD PhDa Send email to raffaella.casolino@glasgow.ac.uk ∙ Joaquin Mateo, MD PhDc ∙ Prof Elisabeth G E de Vries, MDd ∙ Amber Johns, BMedSci PhDa ∙ Melanie Courtot, PhDe,f,g ∙ Prof Rita T Lawlor, PhDj ∙ et al. https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(26)00302-5/abstract?dgcid=raven_jbs_etoc_feature_lanonc

Chemotherapy with or without pembrolizumab followed by maintenance pembrolizumab with or without olaparib as first-line treatment of patients with advanced BRCA non-mutated epithelial ovarian cancer (ENGOT-OV43/GOG-3036/KEYLYNK-001): a randomised, double-blind, placebo-controlled, phase 3 trial Prof Ignace Vergote, MD PhDa,* Send email to ignace.vergote@kuleuven.be ∙ Prof David Cibula, MD PhDb ∙ Prof Matthew Powell, MDc ∙ Annouschka Laenen, PhDa ∙ Prof Els Van Nieuwenhuysen, MD PhDa ∙ Prof Samet Topuz, MDd ∙ et al.

Chemotherapy with or without pembrolizumab followed by maintenance pembrolizumab with or without olaparib as first-line treatment of patients with advanced BRCA non-mutated epithelial ovarian cancer (ENGOT-OV43/GOG-3036/KEYLYNK-001): a randomised, double-blind, placebo-controlled, phase 3 trial Prof Ignace Vergote, MD PhDa,* Send email to ignace.vergote@kuleuven.be ∙ Prof David Cibula, MD PhDb ∙ Prof Matthew Powell, MDc ∙ Annouschka Laenen, PhDa ∙ Prof Els Van Nieuwenhuysen, MD PhDa ∙ Prof Samet Topuz, MDd ∙ et al. https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(26)00223-8/abstract?dgcid=raven_jbs_etoc_feature_lanonc

Microplastics and cancer: a looming threat? The Lancet Oncology (++...)

Microplastics and cancer: a looming threat? The Lancet Oncology https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(26)00483-3/fulltext?dgcid=raven_jbs_etoc_email Oct 2026 Volume 27Number 10p1193-1340, e440-e532 https://www.thelancet.com/journals/lanonc/issue/vol27no10/PIIS1470-2045(26)X2009-5

Derivative of HIV drug could reverse multiple sclerosis symptoms In mice, the new drug led to recovery of motor function and vision by protecting nerve fibers and myelin. Written byAllison Whitten, PhD

Derivative of HIV drug could reverse multiple sclerosis symptoms In mice, the new drug led to recovery of motor function and vision by protecting nerve fibers and myelin. Written byAllison Whitten, PhD https://www.drugdiscoverynews.com/derivative-of-hiv-drug-could-reverse-multiple-sclerosis-symptoms-17556 A new experimental drug could finally turn multiple sclerosis (MS) from a disease that has no cure to one that can be successfully reversed with treatment. Researchers led by Jayakrishna Ambati at the University of Virginia School of Medicine showed that a newly developed chemical derivative of an old HIV drug restored movement and vision in a mouse model of the disease — a feat that no currently approved MS treatment is able to do. The discovery came from an unexpected place. Several years ago, Ambati’s lab began analyzing large health insurance databases and realized that patients taking HIV drugs called nucleoside reverse transcriptase inhibitors (NRTIs) were at a much lower risk of developing the eye disease macular degeneration. Digging in to find out why, the scientists revealed that NRTIs block inflammasome activation that could inhibit the evolution of MS and other chronic diseases.

FDA Approves First Treatment for MCT8 Deficiency First therapy to bypass the broken transporter at the root of this very rare genetic disease and to decrease elevated blood thyroid hormone levels

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-mct8-deficiency The U.S. Food and Drug Administration today approved Emcitate (tiratricol) tablets for oral suspension to treat peripheral thyrotoxicosis (excess thyroid hormone levels in the blood that causes symptoms, such as rapid heart rate, increased blood pressure and adverse effects on metabolism) in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. Emcitate is the first therapy approved by the FDA to treat symptoms of this very rare, devastating genetic disease. This approval reflects the FDA’s dedication to patients with rare genetic diseases, many of whom have serious unmet medical needs.

Can AI help identify communication gaps in cancer care? Siemens Healthineers

https://www.linkedin.com/pulse/can-ai-help-identify-communication-gaps-cancer-care-oiq5f/ A cancer diagnosis can be overwhelming. Patients are suddenly confronted with unfamiliar terminology, treatment options, and a series of appointments that can be difficult to navigate. In this issue, we explore cancer care as an example of a complex patient pathway and ask: How can understanding human experience help improve communication and coordination throughout the care journey? Can AI help? Cancer care often involves multiple departments, specialists, and care teams. While the clinical aspects of care are carefully coordinated, patients may experience a fragmented journey with numerous handovers and interactions across the healthcare system. Communication plays a critical role in this experience, helping patients feel seen, informed, and understood, while also providing care teams with insights that can support more personalized care decisions. Yet information can get lost, questions may remain unanswered, and patients are often required to repeat the same information and concerns at multiple points along their journey. Better understanding these experiences is an important first step toward improving care pathways. As AI tools mature, they may help healthcare organizations identify recurring communication challenges, connect patient feedback to specific moments in the care journey, and support more coordinated care.

Navigating the New Era of mHSPC and nmCRPC Data-Driven Strategies for Therapy Selection CME/CNE/CPE Sunday, 25 October 2026, 13:00 - 14:30 CET MURCIA AUDITORIUM, HALL 3, IFEMA MADRID, MADRID, SPAIN

https://events.medscapelive.org/website/98811/?utm_campaign=462515.01a_esmo_email&utm_source=edu&utm_medium=em&utm_content=ext Join us for an interactive symposium that will transform your approach to prostate cancer treatment. This dynamic 90-minute program brings together leading experts to decode the latest evidence in metastatic hormone-sensitive prostate cancer (mHSPC) and non-metastatic castration-resistant prostate cancer (nmCRPC). Through engaging deep dives and expert roundtable discussions, you'll explore groundbreaking data from pivotal clinical trials and real-world evidence that are reshaping treatment paradigms. Discover how to optimize therapy selection using androgen receptor pathway inhibitors, navigate the complexities of doublet vs triplet therapy, and master risk stratification strategies that put patients first. You'll gain practical insights on balancing efficacy with quality of life, managing treatment-related adverse events, and implementing multidisciplinary care approaches. Walk away with confidence to initiate early intensification strategies that extend survival, delay disease progression, and improve patient outcomes in your clinical practice. IMPORTANT NOTICE To attend this symposium, whether in person or virtually, you must first register directly with ESMO Congress 2026. Please visit the official congress website for full registration details and requirements.

Reshaping Gastroesophageal Adenocarcinoma Care: Integrating New Anti-HER2 Therapies CME/CNE/CPE/IPCE Monday, November 9, 2026 | 15:30 - 16:15 ET Virtual

Reshaping Gastroesophageal Adenocarcinoma Care: Integrating New Anti-HER2 Therapies CME/CNE/CPE/IPCE Monday, November 9, 2026 | 15:30 - 16:15 ET Virtual https://na.eventscloud.com/website/99132/ Reshaping Gastroesophageal Adenocarcinoma Care: Integrating New Anti-HER2 Therapies CME/CNE/CPE/IPCE Monday, November 9, 2026 | 15:30 - 16:15 ET Register Now For seamless registration, login with your Medscape membership account. If you are not logged in, you can register as a guest. Transform your gastroesophageal adenocarcinoma (GEA) practice by joining expert oncologists Elena Elimova, Samuel J. Klempner, and Kohei Shitara for an interactive, 45-minute online discussion. Unpack the latest clinical trial data for novel anti-human epidermal growth factor receptor 2 (HER2) regimens, learn how to integrate new frontline regimens into care for patients with HER2-positive GEA, and gain insight into postprogression strategies. Faculty will also discuss proactive team-based approaches to toxicity management. Register now to elevate your clinical decision-making.

Beyond Chemotherapy Integrating Novel Targeted Agents Into Platinum-Resistant Ovarian Cancer Clinical Practice CME/CNE/CPE Saturday, 24 October 2026, 18:30 - 20:00 CEST PAMPLONA AUDITORIUM, HALL 5, IFEMA MADRID, MADRID, SPAIN

Beyond Chemotherapy Integrating Novel Targeted Agents Into Platinum-Resistant Ovarian Cancer Clinical Practice CME/CNE/CPE Saturday, 24 October 2026, 18:30 - 20:00 CEST PAMPLONA AUDITORIUM, HALL 5, IFEMA MADRID, MADRID, SPAIN https://events.medscapelive.org/website/98737/?utm_campaign=464425.01a_esmo_email&utm_source=edu&utm_medium=em&utm_content=ext Beyond Chemotherapy Integrating Novel Targeted Agents Into Platinum-Resistant Ovarian Cancer Clinical Practice CME/CNE/CPE PAMPLONA AUDITORIUM, HALL 5, IFEMA MADRID, MADRID, SPAIN SATURDAY, 24 OCTOBER 2026 18:30 - 20:00 CEST Register Now Join us in Madrid as our expert faculty address the critical need for advanced therapeutic strategies in platinum-resistant ovarian cancer (PROC). This session will provide clinicians with a comprehensive review of the latest clinical trial evidence surrounding novel antibody–drug conjugates and the safety profiles of emerging immunotherapy–chemotherapy combinations. Participants will enhance their clinical competence in translating biomarker testing results into personalized therapeutic choices. Don’t miss the opportunity to bridge the gap between groundbreaking clinical data and real-world, patient-centered PROC management. IMPORTANT NOTICE To attend this symposium, whether in person or virtually, you must first register directly with ESMO Congress 2026. Please visit the official congress website for full registration details and requirements

Actionable Approaches to AL Amyloidosis Care Diagnosing Earlier, Treating Smarter, Managing Together CME New Orleans Ernest N. Morial Convention Center Friday, December 11, 2026 | 11:30 am - 1:00 pm CT Hybrid

Actionable Approaches to AL Amyloidosis Care Diagnosing Earlier, Treating Smarter, Managing Together CME New Orleans Ernest N. Morial Convention Center Friday, December 11, 2026 | 11:30 am - 1:00 pm CT Hybrid https://na.eventscloud.com/website/98321/ Actionable Approaches to AL Amyloidosis Care Diagnosing Earlier, Treating Smarter, Managing Together CME NEW ORLEANS ERNEST N. MORIAL CONVENTION CENTER FRIDAY, DECEMBER 11, 2026 11:30 AM - 1:00 PM CT Attend In-Person Attend Virtually For seamless registration, login with your Medscape membership account. If you are not logged in, you can register as a guest. In this 90-minute symposium, some of the foremost experts in light-chain (AL) amyloidosis research and care will share actionable strategies for navigating complex challenges in this rare disease. Topics will include Diagnosis, staging, and stratifying: Improving timeliness and accuracy Treatment: Developing personalized, evidence-based plans Patient support: Helping patients navigate the long journey Workflows: Optimizing systems and teamwork The event speakers, involved in some of the most consequential trials in AL amyloidosis, will also examine key data for current treatments and novel approaches like fibril targeting. Register now to secure your spot, and prepare your questions for the live Q&A at the end of the session. Friday Satellite Symposium on Hematology, preceding the 68th ASH Annual Meeting and Exposition

Cracking the Code: Optimizing Outcomes in All Patients With High-Risk HR+/HER2-Negative Early Breast Cancer Authors: Manali Bhave, MD; Shaheenah Dawood, MBBCh, MPH, FACP; Erika Hamilton, MD, FASCO

https://www.medscape.org/viewarticle/cracking-code-optimizing-outcomes-all-patients-high-risk-hr-2026a1000crj?page=1&src=mkmcmr_driv_stan_mscpedu_260928-OUS-HONC-cracking-code-optimizing-outcomes-all-patients-high-risk-hr-2026a1000crj-cta&uac=148436CN

Personalized Cancer Vaccines: What Challenges Remain?

https://www.medscape.com/viewarticle/personalized-cancer-vaccines-what-challenges-remain-2026a1001025?ecd=WNL_trdalrt_pos1_ous_260929_etid8741825&uac=148436CN&impID=8741825 Personalized cancer vaccines are generating renewed interest as new clinical data emerge for melanoma and head and neck cancers. These vaccines target neoantigens selected from mutations specific to each patient’s tumor. In melanoma, Moderna’s messenger RNA (mRNA) vaccine intismeran, combined with pembrolizumab, met the primary endpoint of recurrence-free survival and a key secondary endpoint of distant metastasis-free survival in a phase 3 trial. In head and neck cancer, the personalized vaccine TG4050 induced durable immune responses in a phase 1 trial, although its efficacy still needs to be confirmed.

Sharing the Decision of Treatment Planning in High-Risk HR+/HER2- Early Breast Cancer Authors: Shaheenah Dawood, MBBCh, MPH, FACP

https://www.medscape.org/viewarticle/sharing-decision-treatment-planning-high-risk-hr-her2-early-2026a1000f67?page=1&src=mkmcmr_driv_stan_mscpedu_260929-OUS-HONC-sharing-decision-treatment-planning-high-risk-hr-her2-early-2026a1000f67-cta&uac=148436CN

Forgetting names and losing keys may signal something other than dementia (+...+ +...+)

Forgetting names and losing keys may signal something other than dementia https://www.foxnews.com/health Dr. Tommy Wood breaks down memory's three neurological stages and why noticing your own forgetfulness is actually a good sign, not an Alzheimer's flag. Pope Leo visits site of miracle cures, meets people whose unexplained healings stunned doctors Pope prayed at Massabielle Grotto Saturday night before leading open-air Mass for hundreds of thousands Dr. Marc Siegel By Dr. Marc Siegel Fox News Published September 27, 2026 5:00pm EDT https://www.foxnews.com/health/pope-leo-visits-site-miracle-cures-meets-people-unexplained-healings-stunned-doctors Lobster roll war erupts in New England over controversial ingredient in 'die-hard' divide https://www.foxnews.com/lifestyle?lid=w5cjbglrh3h1 81-year-old refuses to stop working as wife battles cancer, then strangers try to help Texas man had been delivering lost luggage as independent contractor to cover medical expenses for wife By Kelly McGreal Fox News Published September 27, 2026 1:06pm EDT https://www.foxnews.com/health/81-year-old-refuses-stop-working-wife-battles-cancer-strangers-try-help

Navigating Next Steps in CLL: A Collaborative Dialogue Authors: Chaitra Ujjani, MD

https://www.medscape.org/viewarticle/navigating-next-steps-cll-collaborative-dialogue-2026a1000i1d?page=1&src=mkmcmr_driv_stan_mscpedu_260928-OUS-HONC-navigating-next-steps-cll-collaborative-dialogue-2026a1000i1d-cta&uac=148436CN

Could scorpion venom inspire new treatments for liver disease?

https://www.news-medical.net/news/20260908/Could-scorpion-venom-inspire-new-treatments-for-liver-disease.aspx?utm_source=azonetwork_newsletter&utm_medium=email&utm_campaign=drug_discovery_newsletter_18_september_2026 From blocking inflammatory immune pathways to disrupting hepatitis viruses, scorpion-derived peptides are attracting attention as potential drug candidates, but how close are they to becoming clinically useful therapies? In a recent review published in the journal iLIVER, researchers summarized the therapeutic potential, mechanisms, structural classes, applications, limitations, and future development of scorpion venom peptides for inflammatory and hepatic disorders.

Kangaroo care helps smaller babies. Now researchers are studying babies born at normal weight

https://www.news-medical.net/news/20260927/Kangaroo-care-helps-smaller-babies-Now-researchers-are-studying-babies-born-at-normal-weight.aspx Kangaroo mother care is well established for small and preterm babies, but an Indian trial is testing whether extending skin-to-skin contact could also benefit newborns of normal birthweight. A recent paper published in the British Medical Journal Open describes the protocol for a randomized controlled trial examining the benefits of Kangaroo Mother Care (KMC) for babies born at normal birthweight. The study involves three centers in Uttar Pradesh, India.

Researchers mine cobra toxins to uncover a membrane-disrupting antimicrobial peptide

https://www.news-medical.net/news/20260917/Researchers-mine-cobra-toxins-to-uncover-a-membrane-disrupting-antimicrobial-peptide.aspx A computational search of cobra cardiotoxins put 14 venom-inspired peptides through structural, antibacterial, and toxicity testing, providing a rigorous test of how far AI-guided peptide discovery can go. A recent study in the journal NPJ Drug Discovery identified and evaluated antimicrobial peptide candidates derived from cobra cardiotoxins using computational mining, machine learning prioritization, and experimental screening to test a toxin-inspired route to antimicrobial drug discovery.

New artificial intelligence tool identifies aging patterns in hematopoietic stem cells

https://www.news-medical.net/news/20260928/New-artificial-intelligence-tool-identifies-aging-patterns-in-hematopoietic-stem-cells.aspx Aging progressively affects the functioning of our body and, among other things, deteriorates the ability of the hematopoietic system- the set of organs and tissues responsible for producing blood cells- to maintain adequate blood cell production. Understanding and measuring this process is especially relevant to study how blood stem cells age and to identify strategies to preserve or recover their function.

Semaglutide cuts major heart events, but researchers still cannot fully explain why

https://www.news-medical.net/news/20260927/Semaglutide-cuts-major-heart-events-but-researchers-still-cannot-fully-explain-why.aspx A detailed analysis of the SELECT trial examined how much of the reduction in major heart events could be attributed to changes in weight, inflammation, blood glucose, and other cardiovascular risk factors. In a recent study published in the European Heart Journal, researchers investigated which measured cardiovascular risk factors might help explain semaglutide's cardioprotective effects. The study comprised a pre-specified mediation analysis of the landmark SELECT trial, which evaluated 17,604 adults with overweight or obesity and established cardiovascular disease (CVD) without diabetes. Its primary objective was to estimate how much of the drug’s benefit could be explained by improvements in traditional risk factors.

Exam stress significantly raises blood sugar levels in medical students

https://www.news-medical.net/news/20260927/Exam-stress-significantly-raises-blood-sugar-levels-in-medical-students.aspx Exam stress is associated with significant increases in blood sugar levels in medical students both during exams and the days leading up to them, according to new research being presented at the Annual Meeting of The European Association for the Study of Diabetes (EASD) in Milan, Italy (Sept 28 – Oct 2). The findings highlight the measurable metabolic impact of acute psychological stress.

A survey of 371,000 adults links heart disease to eating whole fruit less often

https://www.news-medical.net/news/20260928/A-survey-of-371000-adults-links-heart-disease-to-eating-whole-fruit-less-often.aspx A survey of more than 370,000 US adults compares whole fruit and juice habits across cardiovascular diagnoses, with a closer look at which differences persist after adjustment. In a recent study published in the journal Current Developments in Nutrition, researchers examined differences in the frequency of whole-fruit and 100% fruit-juice consumption between individuals with and without cardiovascular disease (CVD) or CVD risk factors in the United States (US).

New EASD study highlights red meat sugar as independent risk factor for type 2 diabetes

https://www.news-medical.net/news/20260928/New-EASD-study-highlights-red-meat-sugar-as-independent-risk-factor-for-type-2-diabetes.aspx Preliminary results of an observational study being presented at the Annual Meeting of The European Association for the Study of Diabetes (EASD) in Milan, Italy (Sept 28 – Oct 2), reveal that a sugar called dietary N-glycolylneuraminic acid (Neu5Gc), which is common in red meat, is independently associated with a 63% higher risk of developing type 2 diabetes (T2D) in those with the highest versus lowest consumption, even after taking into account other T2D risk factors such as level of physical activity, overall diet, smoking status and other health conditions.

Continuous glucose monitors show strong survival benefits for type 2 diabetes patients

https://www.news-medical.net/news/20260928/Continuous-glucose-monitors-show-strong-survival-benefits-for-type-2-diabetes-patients.aspx People with type 2 diabetes (T2D) on basal insulin, starting continuous glucose monitoring (CGM) had a 44% lower risk of dying from any cause after one year compared with those who did not use CGM, with the risk being 35% lower after two years, according to an analysis of patient data being presented at the Annual Meeting of The European Association for the Study of Diabetes (EASD) in Milan, Italy (Sept 28 – Oct 2). The findings provide the first evidence of an association between advanced CGM technologies and lower risk of death in people living with T2D receiving therapy with basal insulin with or without noninsulin diabetes medications, and also show a reduced risk of cardiovascular events, say researchers.

Research makes strong case for broader use of GLP1 drugs to protect hearts

https://www.news-medical.net/news/20260928/Research-makes-strong-case-for-broader-use-of-GLP1-drugs-to-protect-hearts.aspx New research being presented at the annual meeting of the European Association for the Study of Diabetes (EASD) in Milan, Italy (Sept 28 – Oct 2) makes a case for extending the indication for GLP1- receptor agonists (GLP1-RAs) to millions more people, to protect their heart health. The GLP1-RA drugs semaglutide and tirzepatide are approved for weight loss in adults with a BMI of ≥30 or a BMI of ≥27 plus at least one weight-related health condition e.g. dyslipidaemia (unhealthy levels of cholesterol or other fats in the blood), high blood pressure, type 2 diabetes or previous cardiovascular disease.

Study links insomnia to higher stroke and hospitalization risks

https://www.news-medical.net/news/20260928/Study-links-insomnia-to-higher-stroke-and-hospitalization-risks.aspx Insomnia is associated with a 26% higher risk of stroke and 28% higher odds of hospital admission, according to new research in Sleep Medicine Reviews. The researchers say the findings support recognizing insomnia as an important marker of brain, mental and general health. The review, funded by the European Academy of Neurology (EAN), brings together evidence across seven health outcomes. Alongside the increased risk of stroke and hospitalization, the researchers found that insomnia was associated with depression and suicidal behaviours, while evidence suggested a link with dementia, particularly Alzheimer's disease.

New diabetes pill orforglipron achieves uniform weight loss for women of all ages

https://www.news-medical.net/news/20260928/New-diabetes-pill-orforglipron-achieves-uniform-weight-loss-for-women-of-all-ages.aspx New analyses to be presented at the Annual Meeting of The European Association for the Study of Diabetes (EASD) in Milan, Italy (Sept 28 – Oct 2) show that the new daily oral weight loss / type 2 diabetes medication orforglipron was associated with similar weight loss for women of different ages and at different life stages – premenopausal, experiencing menopause, or post-menopausal. The study is by Dr Hunter Thomas Hoffman, Eli Lilly and Company, Indianapolis, IN, USA – the sponsor of the study and manufacturer of orforglipron – and colleagues. Obesity, a chronic progressive disease, is exacerbated by oestrogen deficiency during menopause. Orforglipron, an oral small-molecule, non-peptide GLP-1 receptor agonist, was associated with significant body weight (BW) reductions in ATTAIN-1 and -2 studies, one of which looked at people living with overweight or obesity but not diabetes (ATTAIN-1) and the other with both conditions (ATTAIN-2). This additional analysis evaluates orforglipron's effect on BW in women by menopausal stage.

New book examines how digital screens cause social disconnection

https://www.news-medical.net/news/20260928/New-book-examines-how-digital-screens-cause-social-disconnection.aspx The most dramatic shift in human communication patterns in history is underway – the migration of social life onto screens – and researchers have issued stark warnings about the long-term impact on the health of our social species. Despite the promise of connection, digital life has produced its opposite, warns Professor of Sociology Vidar Halldorsson. Instead, he says it is reshaping our brains, our communities and our capacity for empathy. "Social media platforms, algorithmic feeds, and digital interfaces increasingly mediate relationships, replacing human-to-human interaction with human-to-object engagement. The implications are not just cultural or technological; they are profoundly sociological," he explains, in his new book How Digital Screen Use Alienates Society.

Childhood cancer survivors face severe mental health challenges as adults

https://www.news-medical.net/news/20260928/Childhood-cancer-survivors-face-severe-mental-health-challenges-as-adults.aspx

New framework maps the complex link between psychological status and cardiovascular health

https://www.news-medical.net/news/20260928/New-framework-maps-the-complex-link-between-psychological-status-and-cardiovascular-health.aspx CVD remains the leading cause of death worldwide, while the global burden of mental disorders continues to rise. Depression and anxiety are associated with myocardial infarction, stroke, heart failure, and cardiovascular mortality, and patients with CVD face a higher risk of comorbid mental disorders. Despite this bidirectional relationship, most studies have examined single diseases or single mechanisms, leaving a fragmented picture of how the brain and heart communicate. Given these challenges, in-depth research is needed on the integrated mechanisms, biomarkers, and interventions that link psychological status and cardiovascular health.

Revealing Proteome Diversity with timsTOF: From Single-Cell Heterogeneity to Non-Canonical Biology

https://www.news-medical.net/webinar/Revealing-Proteome-Diversity-with-timsTOF-From-Single-Cell-Heterogeneity-to-Non-Canonical-Biology?referrer=AZoNFT3&utm_source=azonetwork_newsletter&utm_medium=email&utm_campaign=drug_discovery_newsletter_18_september_2026 Precision medicine assumes an individual's proteome can point physicians toward the right diagnosis and treatment - but that idea rests on recognizing heterogeneity, and single-cell technologies are now revealing heterogeneity within a single cell type itself, reshaping how we understand organs like the heart. In this webinar, Jennifer Van Eyk and Nikolai Slavov explore how single-cell proteomics is uncovering biology invisible to bulk analysis. Van Eyk will share how single-cell proteomics of cardiomyocytes revealed unexpected subpopulations with distinct protein expression, unpredictable drug responsiveness, and cell-environment effects on disease-driving mutations, work now being integrated with spatial omics to map cardiac tissue architecture.

Can Lack of Sleep Change Your Gut Microbiome?

https://www.news-medical.net/health/Can-Lack-of-Sleep-Change-Your-Gut-Microbiome.aspx From disrupted circadian rhythms to changes in microbial metabolites and intestinal barrier function, research is uncovering a close connection between how we sleep and the microorganisms living in our gut. The gut microbiota comprises a diverse community of bacteria, fungi, viruses, archaea, protozoa, and other microorganisms in the gastrointestinal tract, whereas the gut microbiome more broadly encompasses these microorganisms, their genetic material, and their functions.4,5 These microorganisms are involved in digestion, metabolism, and immune regulation. This article explores how sleep disruptions can alter the gut microbiome and its potential impact on metabolic, immune, and mental health.

Combinatorial miRNA signature curbs tumor growth in aggressive cancers New findings slowed tumor growth in a mouse model of triple-negative breast cancer, offering a new template for RNA drug design. Written byAndrea Corona

Combinatorial miRNA signature curbs tumor growth in aggressive cancers New findings slowed tumor growth in a mouse model of triple-negative breast cancer, offering a new template for RNA drug design. Written byAndrea Corona https://www.drugdiscoverynews.com/combinatorial-mirna-signature-curbs-tumor-growth-in-aggressive-cancers-17555 A team at Université Paris Cité has spent six years chasing an unusual observation: Tiny particles released by certain breast cancer cells seemed to make aggressive tumors behave less aggressively. Their new study, published in Molecular Therapy, identifies what's inside those particles that does the work, and offers a possible blueprint for building RNA-based cancer drugs around combinations of molecules rather than single targets. The story started in 2020, when the group, led by molecular biologist Sébastien Jauliac, reported that extracellular vesicles (EVs) released by breast cancer cells expressing a transcription factor called NFAT3 could reduce invasion and metastasis in aggressive cancer models. That raised an obvious follow-up question: what was actually inside those vesicles doing the work?

New CRISPR activation platform uncovers genetic drivers of cancer resistance The next-generation tool, called Partita, identified both known and novel genes that contribute to resistance in lymphomas. Written byAllison Whitten, PhD

New CRISPR activation platform uncovers genetic drivers of cancer resistance The next-generation tool, called Partita, identified both known and novel genes that contribute to resistance in lymphomas. Written byAllison Whitten, PhD https://www.drugdiscoverynews.com/new-crispr-activation-platform-uncovers-genetic-drivers-of-cancer-resistance-17550 Even when a cancer diagnosis has cutting edge treatments available, the threat of resistance lingers. Around 90 percent of cancer-related deaths are estimated to be related to cancer drug resistance, whereby tumor cells carry genetic mutations that allow them to resist the drug from the start, or they acquire them during treatment. Unfortunately, it remains difficult for physicians and researchers to identify which genes cause resistance. Now, researchers led by Marco Herold at the Olivia Newton-John Cancer Research Institute have created a new tool that could make it much easier to identify target genes. The team developed Partita, a whole-genome library for murine models that the research team used to examine blood cancers, including resistance against the commonly prescribed drug venetoclax, a B-cell lymphoma-2 (BCL-2) protein inhibitor.

Landmark map of human brain’s gene activity holds clues to Alzheimer’s disease and more The atlas of gene expression in the prefrontal cortex was created using samples from almost 1,500 donors of all ages — from infants to centenarians. By Amanda Heidt

https://www.nature.com/articles/d41586-026-02947-x?utm_source=Live+Audience&utm_campaign=d6020f3200-nature-briefing-daily-20260924&utm_medium=email&utm_term=0_-33f35e09ea-50432164 Researchers have produced the largest map to date of gene activity in the human prefrontal cortex, a brain area that supports planning, decision-making and behavioural and emotional regulation. The atlas draws on donated samples from almost 1,500 people — from infants to centenarians — and the recorded gene activity in more than 6.3 million individual brain cells. The scale of the map will enable the study of neurodegenerative and psychiatric diseases that affect the prefrontal cortex in unprecedented detail.

‘Multifunctional’ brain implant translates speech and gestures in real time A neural device uses artificial intelligence to read brain activity for intended words and gestures simultaneously. By Miryam Naddaf

https://www.nature.com/articles/d41586-026-02895-6?utm_source=Live+Audience&utm_campaign=3701f6dc34-nature-briefing-daily-20260915&utm_medium=email&utm_term=0_-33f35e09ea-50432164 A new brain–computer interface (BCI) is the first of its kind to translate its user’s speech and movement at the same time. The device, tested in two people with forms of paralysis, uses artificial intelligence to convert electrical brain activity into text that appears on screen and to prompt a personalized animated avatar to move — all within seconds of the user’s intent. The work is “a step towards more multifunctional BCIs” that better capture the expressivity of communication than do current technologies, says bioengineer and study co-author Samantha Brosler.

CDRH: Molecular and Clinical Genetics Panel of the Medical Devices Advisory Committee (MDAC)

Meeting Reminder and Materials: Molecular and Clinical Genetics Panel of the Medical Devices Advisory Committee On September 23, 2026 the Molecular and Clinical Genetics Panel of the Medical Devices Advisory Committee (the Committee) will meet in an open session to discuss, make recommendations, and vote on information regarding the premarket approval application (PMA) for the Galleri® test sponsored by GRAIL Inc. The Galleri® test is a prescription-only qualitative, next-generation sequencing (NGS)-based in vitro diagnostic test intended to detect cancer-specific methylation patterns in cell-free DNA isolated from peripheral whole blood. The Galleri® test is intended for screening for the early detection of multiple types of cancer in adults aged 50 years or older. GRAIL Inc. and FDA will present key information related to the PMA application. The meeting is open to the public. Registration is not required to attend the meeting.

Unsafe by design? ‍The launch of the Lancet Series on the digital determinants of health

https://www.dthlab.org/unsafe-by-design?utm_medium=email&_hsenc=p2ANqtz-9fvcsuDOxBiVts_GjHuw2UChT-ayhO4kRQ42YlvJT6cUGrja_Rx_RnYsdduryrMYn5Hi0KLF6AB8_3YMAb9zuG4lv88Q&_hsmi=439865762&utm_content=439626183&utm_source=hs_email The Lancet Series on the digital determinants of health launch webinar Oct 5, 2026 13:00 - 14:30 BST We are pleased to invite you to the free launch webinar of The Lancet Series on the digital determinants of health. The webinar will be moderated by Richard Horton, Lancet Editor-in-Chief, and Ben Abbott, Lancet Executive Editor, and co-hosted by DTH-Lab. Bringing together Series authors, young people, public health leaders, policymakers and governance experts, the webinar will examine why digital environments should be treated as a public health priority and how we can move from responding to harm to preventing it.

How to make a brain: new experiments challenge existing picture Research also uncovers an efficient way to grow hindbrain cells from stem cells — a difficult feat. By Lynne Peeples

https://www.nature.com/articles/d41586-026-02943-1?utm_source=Live+Audience&utm_campaign=896a49cb62-nature-briefing-daily-20260921&utm_medium=email&utm_term=0_-33f35e09ea-50432164 A new study suggests that two types of precursor cells are required to build the brain, challenging a decades-long theory that a single type of starter cell gives rise to the complex organ. The research finds that one type of precursor cell forms the hindbrain, while another generates the forebrain and midbrain. Not all researchers are convinced by this conclusion, but the work has drawn broad praise for one aspect: the authors found an efficient way to coax stem cells to grow into hindbrain motor-neuron cells, which could aid research into conditions that affect these cells, such as amyotrophic lateral sclerosis (ALS).

How to stay smart in the age of AI: the science of critical thinking There is growing concern that AI can blunt memory and reasoning. But science shows ways to keep the brain sharp. By Helen Pearson

https://www.nature.com/articles/d41586-026-02930-6?utm_source=Live+Audience&utm_campaign=bc19c8fc46-nature-briefing-daily-20260926&utm_medium=email&utm_term=0_-33f35e09ea-50432164

All the ways a cell can die — and why the variety matters Scientists are poking holes in established ideas about how cells live and die, and are harnessing these discoveries to fight diseases from cancer to autoimmune conditions. By Amber Dance

https://www.nature.com/articles/d41586-026-02929-z?utm_source=Live+Audience&utm_campaign=bc19c8fc46-nature-briefing-daily-20260926&utm_medium=email&utm_term=0_-33f35e09ea-50432164

Community-acquired pneumonia in adults: acute and long-term complications Jodie Chalmers, MB BChira,b Send email to Jodie.chalmers@bristol.ac.uk ∙ Krishan Bansal, MB BChirb ∙ Rachel Scott, MB BChira,b ∙ Fergus Hamilton, PhDc ∙ Prof Rupert Payne, PhDd ∙ Prof Wei Shen Lim, MDe ∙ et al.

Community-acquired pneumonia in adults: acute and long-term complications Jodie Chalmers, MB BChira,b Send email to Jodie.chalmers@bristol.ac.uk ∙ Krishan Bansal, MB BChirb ∙ Rachel Scott, MB BChira,b ∙ Fergus Hamilton, PhDc ∙ Prof Rupert Payne, PhDd ∙ Prof Wei Shen Lim, MDe ∙ et al. https://www.thelancet.com/journals/lanhl/article/PIIS2666-7568(26)00056-5/fulltext?dgcid=raven_jbs_etoc_feature_lanhl

Frailty, clinical outcomes, and treatment effects of a care bundle in acute intracerebral haemorrhage: a post-hoc analysis of the INTERACT3 trial Tao Liu, MDa,∗ ∙ Linan Chen, PhDa,∗ ∙ Yang Liu, MDb ∙ Zhihao Zhao, MDb ∙ Leibo Liu, PhDa ∙ Prof Lu Ma, MDc ∙ et al.

Frailty, clinical outcomes, and treatment effects of a care bundle in acute intracerebral haemorrhage: a post-hoc analysis of the INTERACT3 trial Tao Liu, MDa,∗ ∙ Linan Chen, PhDa,∗ ∙ Yang Liu, MDb ∙ Zhihao Zhao, MDb ∙ Leibo Liu, PhDa ∙ Prof Lu Ma, MDc ∙ et al. http://thelancet.com/journals/lanhl/article/PIIS2666-7568(26)00073-5/fulltext?dgcid=raven_jbs_etoc_feature_lanhl

Ageing Well for Indigenous Peoples: Closing the Health Equity Gap The Lancet Healthy Longevity +...

Ageing Well for Indigenous Peoples: Closing the Health Equity Gap The Lancet Healthy Longevity https://www.thelancet.com/journals/lanhl/issue/vol7no8/PIIS2666-7568(26)X2008-6

Effect of a community-based behavioural intervention bundle to improve antibiotic use and patient management in Burkina Faso and DR Congo (CABU-EICO): a cluster-randomised controlled trial

Effect of a community-based behavioural intervention bundle to improve antibiotic use and patient management in Burkina Faso and DR Congo (CABU-EICO): a cluster-randomised controlled trial Dr Brecht Ingelbeen, PhDa,* Send email to bingelbeen@itg.be ∙ Daniel Valia, PhDb,* ∙ Bijou Mbangi, MDc ∙ Esther van Kleef, PhDd,e ∙ Linda Campbell, PhDf ∙ Juste Stéphane Kouanda, MScb ∙ et al. https://www.thelancet.com/journals/laninf/article/PIIS1473-3099(26)00169-6/abstract?dgcid=raven_jbs_etoc_feature_laninf

Effectiveness of oral care for the prevention of non-ventilator hospital-acquired pneumonia (HAPPEN): a multicentre, stepped-wedge, cluster-randomised trial in Australia

Effectiveness of oral care for the prevention of non-ventilator hospital-acquired pneumonia (HAPPEN): a multicentre, stepped-wedge, cluster-randomised trial in Australia Nicole M White, PhDa ∙ Prof Philip L Russo, PhDb,c,d,j ∙ Georgia Matterson, Biotech(Hons)d ∙ Katrina Browne, PhDe ∙ Prof Allen C Cheng, MBBSf ∙ Martin Kiernan, MClinResd,g ∙ et al. https://www.thelancet.com/journals/laninf/article/PIIS1473-3099(26)00235-5/fulltext?dgcid=raven_jbs_etoc_feature_laninf

Counteracting the rise of antimicrobial resistance in Shigella The Lancet Infectious Diseases +...

Counteracting the rise of antimicrobial resistance in Shigella The Lancet Infectious Diseases https://www.thelancet.com/journals/laninf/issue/vol26no10/PIIS1473-3099(26)X2009-6

Interprofessional Approach to Adverse Event Care for the Patient With High-Risk Hormone Receptor-Positive/HER2-Negative Early Breast Cancer Authors: Jonathan Malara, PharmD, BCOP; Nelli Zafman, CRNP, MSN, AOCNP

https://www.medscape.org/viewarticle/interprofessional-approach-adverse-event-care-patient-high-2026a1000gez?page=1&src=mkmcmr_driv_stan_mscpedu_260926-OUS-HONC-interprofessional-approach-adverse-event-care-patient-high-2026a1000gez-cta&uac=148436CN

Expert Panel Decision Framework: Progressive Strategies for Managing HER2-Mutated Advanced NSCLC Authors: Charu Aggarwal, MD, MPH; Herbert H. Loong, MBBS, FRCP; Umberto Malapelle, PhD

https://www.medscape.org/viewarticle/expert-panel-decision-framework-progressive-strategies-2026a1000diy?page=1&src=mkmcmr_driv_stan_mscpedu_260927-OUS-HONC-expert-panel-decision-framework-progressive-strategies-2026a1000diy-cta&uac=148436CN

Sep 04, 2026 This Week in Cardiology Podcast John M. Mandrola, MD +++

https://www.medscape.com/viewarticle/1003510?ecd=mkm_mscpapp_260926_mscpmrk_podcasts_etid8728255&uac=148436CN&impID=8728255 Choosing Therapies for ATTR-CM in 2026 Kevin M. Alexander, MD; Marcus A. Urey, MD https://www.medscape.com/ca8/p04/podcast-attr-cm-s1-ep6-2026a10006hh?ecd=mkm_mscpapp_260926_mscpmrk_podcasts_etid8728255&uac=148436CN&impID=8728255 How Early Is Early Enough to Reverse MASLD? Lisa B. VanWagner, MD, MSc; Vincent L. Chen, MD https://www.medscape.com/ca8/p04/podcast-masld-s1-ep4-2026a1000aaw?ecd=mkm_mscpapp_260926_mscpmrk_podcasts_etid8728255&uac=148436CN&impID=8728255

What Can Risk Factors Teach Us About Endometrial Cancer? InDiscussion Endometrial Cancer Ursula A. Matulonis, MD; Immaculata De Vivo, MPH, PhD

https://www.medscape.com/ca8/p04/podcast-endometrial-cancer-s3-ep6-2026a10006ig?ecd=mkm_mscpapp_260926_mscpmrk_podcasts_etid8728255&uac=148436CN&impID=8728255

Gout and Comorbidities: A Two-Way Relationship Kenneth Saag, MD; Michael H. Pillinger, MD

https://www.medscape.com/ca8/p04/podcast-gout-s1-ep1-2026a1000qeh?ecd=mkm_mscpapp_260926_mscpmrk_podcasts_etid8728255&uac=148436CN&impID=8728255

Steps for Personalized and Collaborative Decision-Making in HER2-Mutated NSCLC Authors: Pasi Jänne, MD, PhD, FASCO; Soo-Ryum (Stewart) Yang, MD; Xiuning Le, MD, PhD

https://www.medscape.org/viewarticle/steps-personalized-and-collaborative-decision-making-her2-2026a10007ur?page=1&src=mkmcmr_driv_stan_mscpedu_260927-OUS-HONC-steps-personalized-and-collaborative-decision-making-her2-2026a10007ur-cta&uac=148436CN

Radiotherapy versus observation following surgical resection of WHO grade 2 atypical meningioma (ROAM/EORTC-1308): an international, multicentre, open-label, phase 3, randomised controlled trial Prof Michael D Jenkinson, PhDa,b Send email to michael.jenkinson@liverpool.ac.uk ∙ Anna Rosala-Hallas, MScc,* ∙ Prof Felix Sahm, PhDd,e,* ∙ Prof Nicolaus Andratschke, MDf ∙ Prof Keyoumars Ashkan, DScg ∙ Damiano Balestrini, MDh ∙ et al.

Radiotherapy versus observation following surgical resection of WHO grade 2 atypical meningioma (ROAM/EORTC-1308): an international, multicentre, open-label, phase 3, randomised controlled trial Prof Michael D Jenkinson, PhDa,b Send email to michael.jenkinson@liverpool.ac.uk ∙ Anna Rosala-Hallas, MScc,* ∙ Prof Felix Sahm, PhDd,e,* ∙ Prof Nicolaus Andratschke, MDf ∙ Prof Keyoumars Ashkan, DScg ∙ Damiano Balestrini, MDh ∙ et al. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01804-0/fulltext?dgcid=hubspot_email_conferencealerts_eano26&utm_campaign=conferencealerts&utm_medium=email&_hsenc=p2ANqtz--e9B_H7XYpluwgPJpsD-oPH2REdwCheor0XexlCSCWqbu0USlZkuVdimTXyNmgV8nAKPHg9do2ZYWZPdVPdHu9B2gZ5Q&_hsmi=440950896&utm_content=440950896&utm_source=hs_email

Same-Day Cancer Clinics: Data Support Team Approach Maurie Markman, MD

https://www.medscape.com/viewarticle/same-day-cancer-clinics-data-support-team-approach-2026a1000xc1?src= Hello. I'm Dr Maurie Markman, from City of Hope. I'd like to discuss a very interesting paper. In these commentaries, I often try to stick to definitive trials and randomized trials. I'm not doing that right here, but I think it's a very important, relevant topic for cancer care today.

Fast Five Quiz: Are You Familiar With Key Aspects of Polycythemia Vera? Emmanuel C. Besa, MD

https://reference.medscape.com/viewarticle/fast-five-quiz-are-you-familiar-key-aspects-polycythemia-2025a1000dnl?_gl=1*7bqmer*_gcl_au*MTA2Mzc5MDEwMS4xNzg5ODMwNjQxLjEyODIyNTQ2NDYuMTc5MDQzMzg5My4xNzkwNDM0ODg2LjExNjk1NDYxNzkuMTc5MDQzMzg5My4xNzkwNDM0ODg2 Polycythemia vera (PV) is a clonal myeloproliferative neoplasm in which increased red-cell mass causes hyperviscosity and raises the risk for arterial and venous thrombosis. Common findings include headache, light-headedness, and visual symptoms. Some patients have bleeding complications.

Heartburn, Epigastric Pain, and Weight Loss in a 63-Year-Old Woman Avan J. Armaghani, MD

https://reference.medscape.com/viewarticle/heartburn-epigastric-pain-and-weight-loss-63-year-old-woman-2026a1000ytd?_gl=1*kve10o*_gcl_au*MTA2Mzc5MDEwMS4xNzg5ODMwNjQxLjEyODIyNTQ2NDYuMTc5MDQzMzg5My4xNzkwNDM0ODg2LjExNjk1NDYxNzkuMTc5MDQzMzg5My4xNzkwNDM0ODg2

What to Know Before Using IM Kenalog Hana Ahmed, MD

https://www.medscape.com/viewarticle/what-know-before-using-im-kenalog-2026a1000yz4?src= Intramuscular triamcinolone acetonide, or IM Kenalog, is a treatment modality that can be used in dermatology but only in very specific and severe circumstances.

Guidelines for the Use of Antiretroviral Agents in Adults and Adolescents With HIV

https://clinicalinfo.hiv.gov/en/guidelines/hiv-clinical-guidelines-adult-and-adolescent-arv/whats-new

Optimizing the Transplant Experience: A Patient Journey Through Conditioning and GvHD Management Authors: Daniele Avenoso, MD, PhD; Filippo Milano, MD, PhD; Mareike Verbeek, MD; Robert Zeiser, MD

https://www.medscape.org/viewarticle/optimizing-transplant-experience-patient-journey-through-2026a1000bh8?page=1&src=mkmcmr_driv_stan_mscpedu_260926-OUS-HONC-optimizing-transplant-experience-patient-journey-through-2026a1000bh8-cta&uac=148436CN

Accelerating equitable cancer genomics and precision oncology in health care and research: a Lancet Oncology Commission Raffaella Casolino, MD PhDa Send email to raffaella.casolino@glasgow.ac.uk ∙ Joaquin Mateo, MD PhDc ∙ Prof Elisabeth G E de Vries, MDd ∙ Amber Johns, BMedSci PhDa ∙ Melanie Courtot, PhDe,f,g ∙ Prof Rita T Lawlor, PhDj ∙ et al.

Accelerating equitable cancer genomics and precision oncology in health care and research: a Lancet Oncology Commission Raffaella Casolino, MD PhDa Send email to raffaella.casolino@glasgow.ac.uk ∙ Joaquin Mateo, MD PhDc ∙ Prof Elisabeth G E de Vries, MDd ∙ Amber Johns, BMedSci PhDa ∙ Melanie Courtot, PhDe,f,g ∙ Prof Rita T Lawlor, PhDj ∙ et al. https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(26)00302-5/abstract?utm_campaign=conferencealerts&utm_medium=email&dgcid=hubspot_email_conferencealerts_wcc26&_hsenc=p2ANqtz-_LRk2iL_CvF18ee5L_VbylL5sAY_bIgcQsUK1x2WlIofW-w54tKi3Q_vxRncy8GGenRY0IEs_SQgHInX4yhGzndqlnwg&_hsmi=440498214&utm_content=440498214&utm_source=hs_email

Influenza Vaccine Linked to Fewer Influenza-Related Hospitalizations in COPD, but Confounding Remains a Concern September 24, 2026

https://www.medscape.com/viewarticle/influenza-vaccine-linked-fewer-influenza-related-2026a1000zmw?ecd=wnl_tp10_daily_260926_MSCPEDIT_etid8728689&uac=148436CN&impID=8728689 Influenza vaccination was associated with a 39% lower risk for hospitalization for influenza among patients with chronic obstructive pulmonary disease (COPD) during epidemic periods across six influenza seasons in England. However, vaccination was also linked to a 17% lower risk for urinary tract infection, which served as a negative control outcome with no plausible causal connection to the vaccine, suggesting that residual confounding may have biased the findings.

Beyond Lifestyle: A Practical Guide to Liver-Directed Therapy for MASH photo of a inflamed liver Lisa B. VanWagner, MD, MSc; Mazen Noureddin, MD, MHSc

https://www.medscape.com/ca8/p04/podcast-masld-s1-ep5-2026a1000aax?ecd=wnl_tp10_daily_260926_MSCPEDIT_etid8728689&uac=148436CN&impID=8728689

Tirzepatide: Modest Weight Regain, Sustained Glycemic Gains Over 3 Years September 24, 2026

https://www.medscape.com/viewarticle/tirzepatide-modest-weight-regain-sustained-glycemic-gains-2026a1000zo4?ecd=wnl_tp10_daily_260926_MSCPEDIT_etid8728689&uac=148436CN&impID=8728689 In a 3-year analysis of the SURMOUNT-1 trial, most participants with obesity or overweight and prediabetes who reached their lowest weight on tirzepatide had regained little of it by the end of the study — 70% regained less than 5% of their baseline weight. Even with some rebound, patients retained most of their metabolic gains — suggesting a small amount of regain is not necessarily a cause for concern.

What to Know About New MI Definitions Nicholas L. Mills, MBBS

https://www.medscape.com/viewarticle/what-know-about-new-mi-definitions-2026a1000y8p?ecd=wnl_tp10_daily_260926_MSCPEDIT_etid8728689&uac=148436CN&impID=8728689 My name’s Nick Mills. I’m the British Heart Foundation Chair of Cardiology at the University of Edinburgh. We’ve just released the Fifth Universal Definition of Myocardial Infarction. Why is this important for your practice? Well, firstly, the previous numerical classification of myocardial infarction has been replaced.

Gut Microbiome May Shape Hormone-Dependent Cancers Laurence Salmon

https://www.medscape.com/viewarticle/gut-microbiome-may-shape-hormone-dependent-cancers-2026a1000zrw?ecd=wnl_tp10_daily_260926_MSCPEDIT_etid8728689&uac=148436CN&impID=8728689 Key Takeaways In hormone-dependent cancers, the role of the microbiome in estrogen metabolism (the estrobolome) extends to an endocrine-microbiota axis based on reciprocal interactions with the endocrine system. Several mechanisms, including dysbiosis and chronic inflammation, may promote carcinogenesis. Modulation of the microbiome represents a promising — though still experimental — therapeutic approach.

Reshaping Gastroesophageal Adenocarcinoma Care: Integrating New Anti-HER2 Therapies CME/CNE/CPE/IPCE Monday, November 9, 2026 | 15:30 - 16:15 ET

https://na.eventscloud.com/website/99132/ Transform your gastroesophageal adenocarcinoma (GEA) practice by joining expert oncologists Elena Elimova, Samuel J. Klempner, and Kohei Shitara for an interactive, 45-minute online discussion. Unpack the latest clinical trial data for novel anti-human epidermal growth factor receptor 2 (HER2) regimens, learn how to integrate new frontline regimens into care for patients with HER2-positive GEA, and gain insight into postprogression strategies. Faculty will also discuss proactive team-based approaches to toxicity management. Register now to elevate your clinical decision-making.

Childhood Cancers

https://www.cancer.gov/types/childhood-cancers A cancer diagnosis is upsetting at any age, but especially so when the patient is a child. It's natural to have many questions, such as, Who should treat my child? Will my child get well? What does all of this mean for our family? Not all questions have answers, but the information and resources on this page provide a starting point for understanding the basics of childhood cancer.

Understanding Adult Overweight & Obesity

https://www.niddk.nih.gov/health-information/weight-management/adult-overweight-obesity

Antibodies may prevent tick-related syndrome

Antibodies may prevent tick-related syndrome At a Glance Researchers identified rare antibodies that may help combat alpha-gal syndrome, a condition that causes allergic reactions to red meat and dairy products. With further development, the finding could lead to treatments for this tick-related allergic condition. https://www.nih.gov/news-events/nih-research-matters/antibodies-may-prevent-tick-related-syndrome

Addressing ADHD Support for Parents and Kids

https://newsinhealth.nih.gov/2026/09/addressing-adhd

Shaping the Future of Precision Oncology: Biomarker-Driven Care for Tumor-Agnostic Therapies Authors: Benedikt Westphalen, MD, PhD; Vivek Subbiah, M

https://www.medscape.org/viewarticle/shaping-future-precision-oncology-biomarker-driven-care-2026a1000jpk?page=1&src=mkmcmr_driv_stan_mscpedu_260925-OUS-HONC-shaping-future-precision-oncology-biomarker-driven-care-2026a1000jpk-cta&uac=148436CN

A Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Participants With Childhood Onset Idiopathic Nephrotic Syndrome (INShore)

https://clinicaltrials.gov/study/NCT05627557?utm_medium=email&utm_source=govdelivery FDA Approves Drug to Treat Idiopathic Nephrotic Syndrome in Patients 2 Years and Older Action FDA has approved Gazyva (obinutuzumab) injection to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or steroid-dependent, childhood-onset, idiopathic nephrotic syndrome who are in remission. Gazyva was previously approved for treating certain cancers and for treating adults with active lupus nephritis who are receiving standard therapy. Disease or Condition Idiopathic nephrotic syndrome is a kidney disorder of unknown cause. In these patients, large amounts of protein leak into the urine, leading to low blood albumin levels (a type of protein in the blood) and often swelling. Idiopathic nephrotic syndrome can also lead to high cholesterol and other complications such as infections, and blood clots that can block small blood vessels. Idiopathic nephrotic syndrome is the most common cause of nephrotic syndrome in children and usually starts between 2-7 years of age. The annual incidence is estimated to be 1 to 4 per 100,000 children and varies with age, race, and geography. Effectiveness The efficacy and safety of Gazyva were evaluated in INSHORE (NCT05627557), a phase 3, randomized, controlled, open-label, multicenter study in 85 patients 2 years of age and older with childhood-onset frequently relapsing or steroid-dependent idiopathic nephrotic syndrome. Patients had to be in complete remission at study entry, meaning no swelling and urine protein within the normal range. In this study, patients were randomly assigned to receive intravenous Gazyva dosed according to the patient’s weight on Days 1 and 15, and Weeks 24 and 26, or twice daily oral mycophenolate mofetil, a common standard of care therapy. The primary endpoint was the proportion of patients with first morning urine protein-creatinine ratio (a measure of the amount of protein in the urine) of no more than 0.2 g/g at Week 52 without a relapse from Week 8 to Week 52. The proportion of patients meeting this endpoint was significantly greater in the Gazyva treatment group compared to the standard of care treatment group (73%). Safety Gazyva’s label has a boxed warning for hepatitis B virus reactivation and progressive multifocal leukoencephalopathy (a rare and serious brain infection caused by a virus in people with a weakened immune system). The most common side effects of Gazyva in patients with idiopathic nephrotic syndrome are infections, infusion-related reactions, and neutropenia (low number of a type of white blood cell that fights infections). See the prescribing information for the complete safety information. Gazyva received Breakthrough Therapy, Orphan Drug and Priority Review designations for this approval.

Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial Maja Di Rocco 1, Eduardo Forleo-Neto 2, Robert J Pignolo 3, Richard Keen 4, Philippe Orcel 5 6, Thomas Funck-Brentano 5 6, Christian Roux 7, Sami Kolta 7, Annalisa Madeo 1, Judith S Bubbear 4, Jacek Tabarkiewicz 8, Małgorzata Szczepanek 8, Javier Bachiller-Corral 9, Angela M Cheung 10, Kathryn M Dahir 11, Esmée Botman 12, Pieter G Raijmakers 13, Mona Al Mukaddam 14, Lianne Tile 10, Cynthia Portal-Celhay 2, Neena Sarkar 2, Peijie Hou 2, Bret J Musser 2, Anita Boyapati 2, Kusha Mohammadi 2, Scott J Mellis 15, Andrew J Rankin 2, Aris N Economides 2, Dinko Gonzalez Trotter 2, Gary A Herman 2, Sarah J O'Meara 2, Richard DelGizzi 2, David M Weinreich 2, George D Yancopoulos 2, E Marelise W Eekhoff # 12, Frederick S Kaplan

Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial Maja Di Rocco 1, Eduardo Forleo-Neto 2, Robert J Pignolo 3, Richard Keen 4, Philippe Orcel 5 6, Thomas Funck-Brentano 5 6, Christian Roux 7, Sami Kolta 7, Annalisa Madeo 1, Judith S Bubbear 4, Jacek Tabarkiewicz 8, Małgorzata Szczepanek 8, Javier Bachiller-Corral 9, Angela M Cheung 10, Kathryn M Dahir 11, Esmée Botman 12, Pieter G Raijmakers 13, Mona Al Mukaddam 14, Lianne Tile 10, Cynthia Portal-Celhay 2, Neena Sarkar 2, Peijie Hou 2, Bret J Musser 2, Anita Boyapati 2, Kusha Mohammadi 2, Scott J Mellis 15, Andrew J Rankin 2, Aris N Economides 2, Dinko Gonzalez Trotter 2, Gary A Herman 2, Sarah J O'Meara 2, Richard DelGizzi 2, David M Weinreich 2, George D Yancopoulos 2, E Marelise W Eekhoff # 12, Frederick S Kaplan https://pubmed.ncbi.nlm.nih.gov/37770652/ FDA Approves Third Treatment for Fibrodysplasia Ossificans Progressiva Action The U.S. Food and Drug Administration (FDA) has approved Atebrioz (zilurgisertib) tablets to reduce the volume of total new heterotopic ossification (bone formation outside the skeleton) in adults and pediatric patients 12 years and older with fibrodysplasia ossificans progressiva (FOP). The recommended starting dosage is 100 mg orally once daily with or without food. Disease or Condition Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease caused by a mutation in activin A receptor-type 1, which controls new bone growth. As a result, connective tissues such as muscle, tendons, and ligaments gradually turn into bone, causing limited movement, deformities, severe disability, and early death. Effectiveness The effectiveness of Atebrioz was evaluated in a randomized, double-blind, placebo controlled trial (NCT05090891) in which 63 patients with FOP were randomly assigned to receive Atebrioz 100 mg or placebo once daily for 24 weeks followed by a 292-week, single-arm, open-label extension period during which patients received Atebrioz 100 mg daily. Atebrioz’s efficacy was based on the change from baseline in volume of total new heterotopic ossification compared to placebo during the double blind period, assessed by whole body CT scans. At Week 24, the group of patients receiving Atebrioz had an average 3.2 cm³ decrease in volume of total new heterotopic ossification, whereas the placebo group had a 24.6 cm³ increase. Safety Information Atebrioz can cause fetal harm based on data from animal studies. Patients of reproductive potential should use effective contraception and should immediately discontinue Atebrioz and contact their healthcare provider if pregnancy occurs. The most common side effects are headache, joint pain, upper respiratory tract infection, nosebleeds, and nausea. Atebrioz should not be taken with certain other medications, as described in the label. Designations Atebrioz received fast track, priority review and orphan drug designation for this indication.

The gene-regulatory evolution of the human skeleton Yizhi Yan, Nadav Mishol, Katharina Lange, Zicong Zhang, Gal Bodek, Aya Kigel, Noam Priel, Nachshon Egyes, Omer Ronen, Itamar Nini, Liat Rotenstreich, Amit Philosoph, Sira Martinez, Silvia Beltramone, Rika Tsujikawa, Adi Rozenblatt, Lucas Esteban Wange, María Torralvo, Guy Hirsh, Yael Elboim, Sergey Viukov, Idan Korenfeld, Mythili Damal Kandadai, Océane Cluzeau, …David Gokhman

https://www.nature.com/articles/s41586-026-11053-x Skeletal modifications were central to human evolution, enabling adaptations for bipedalism, large cranial vaults and childbirth1. Despite their importance, the genetic changes that gave rise to the unique human form remain mostly unknown2. Here we systematically map the gene-regulatory changes that shaped human skeletal evolution. Using massively parallel reporter assays (MPRAs) in chondrocytes, we assayed 561,410 human-derived substitutions in promoters and enhancers, identifying 15,077 loci with human-specific regulatory activity. We then generated human–ape hybrid cells and differentiated them into osteochondral progenitors. Integrating the hybrid cells with MPRA measurements produced genome-wide atlases of human-specific changes in cis-regulatory expression, and the sequence variants that drive them. These atlases reveal an extensive rewiring of the extracellular matrix (ECM), including a marked suppression of glycosaminoglycan (GAG) biosynthesis, leading to an approximately three-to-fourfold reduction in joint GAG content in humans compared with non-human apes. We find that this human-specific shift bears signatures of selection, and is likely to be a key contributor to the exceptional susceptibility of humans to degenerative skeletal diseases3,4,5. Together, our results reveal a coordinated evolutionary remodelling of the human skeletal ECM, and establish a comprehensive framework for dissecting the genetic basis of human skeletal biology.

Micelles in cancer nanomedicine: targeted delivery, imaging, and therapy Amirhossein Rashnoodi [1]ORCID iD , Zaynab Seraj [1]ORCID iD , Niyoosha Zamani [1]ORCID iD , Bahareh Khalili Najafabad* [1]

https://www.academia.edu/3071-2521/2/3/10.20935/AcadDrug8458

Pharmacometric generative stochastic modeling of patient-reported outcome measures Kuteesa R. Bisaso* [1] , Karungi S. Bisaso [1] , Karyaburo R. Kadada [1] , Jackson K. Mukonzo [2] , Ene I. Ette [3]

https://www.academia.edu/3071-2521/2/2/10.20935/AcadDrug8393 Introduction: Patient-reported outcome measures (PROMs) capture the patient’s own perspective on their health, illness, and therapeutic effects on the illness. PROM data are inherently high-dimensional, discrete, and interdependent, challenging standard models that rely on restrictive assumptions and often collapse item-level information. This study, therefore, investigates the use of restricted Boltzmann machines (RBM) to jointly model, simulate, and interpret multidimensional PROM data within a pharmacometric framework. Materials and methods: A mixed-variate RBM was applied to longitudinal neuropsychological impairment data from 157 HIV-positive patients receiving efavirenz. The model jointly represented binary symptom items, an ordinal mini-mental state examination (MMSE) measure, efavirenz mid-dose concentrations, and clinical variables (CD4 cell count and viral load) using an energy-based formulation. Parameters were estimated via persistent contrastive divergence. Model performance was evaluated using reconstruction error, pseudolikelihood, free-energy stability, and predictive accuracy on a held-out dataset, alongside simulation-based diagnostics including visual predictive checks. The model was used to derive a variable importance ranking for all the PROM items, clinical variables, and drug concentrations. Results: The RBM captured joint dependencies across PROM items and covariates. On the held-out test set, the model demonstrated good conditional predictive performance, with log-loss ranging from 0.01 to 0.72, Brier scores from 0.00 to 0.26, and mean squared error (MSE) from 0.01 to 0.50. Excellent calibration was achieved at week 12 (t84), particularly for sleepwalking, tactile, and visual hallucinations. Visual predictive checks showed good agreement between observed and simulated symptom trajectories. Variable importance analysis indicated that mid-dose concentrations were not more predictive of post-baseline PROMs than clinical variables and baseline PROMs. Therapeutic drug monitoring simulations revealed limited impact of concentration capping on neuropsychological outcomes. Conclusions: Generative RBMs provide a flexible and minimally assumptive framework for pharmacometric PROM analysis, enabling joint modeling and simulation of complex symptom data. This approach complements traditional methods and supports the use of baseline clinical state over single exposure metrics for prediction. https://www.academia.edu/journals/academia-drug-development-and-pharmacotherapy/articles?source=journal-top-nav

GLP-1 drugs fail to help some people lose weight — scientists are on a quest for answers Understanding why some people see no benefits from potent anti-obesity medications could lead to new therapies and personalized weight-loss regimens. By Mariana Lenharo

https://www.nature.com/articles/d41586-026-03020-3?utm_source=Live+Audience&utm_campaign=5858217fe2-nature-briefing-daily-20260925&utm_medium=email&utm_term=0_-33f35e09ea-50432164 Around 10–15% of people taking popular GLP-1 drugs such as Wegovy lose little to no weight — and researchers don’t really know why. Solving this mystery might lead to new medications for people who don’t benefit from currently available drugs, and could make it possible to match individuals to treatments that are most likely to work for them.

Whole genome sequencing for Klebsiella pneumoniae in the Tunisian antimicrobial resistance surveillance system: micro-costing, pragmatic cost-effectiveness, and budget impact evaluation Dana Itania Send email to dana.itani@alumni.lshtm.ac.uk ∙ Kasim Allelb,c ∙ Sanaa Farjanid,k ∙ Hanen Smaouie ∙ Meriam Zribif ∙ Lamia Thabetg ∙ et al.

Whole genome sequencing for Klebsiella pneumoniae in the Tunisian antimicrobial resistance surveillance system: micro-costing, pragmatic cost-effectiveness, and budget impact evaluation Dana Itania Send email to dana.itani@alumni.lshtm.ac.uk ∙ Kasim Allelb,c ∙ Sanaa Farjanid,k ∙ Hanen Smaouie ∙ Meriam Zribif ∙ Lamia Thabetg ∙ et al. https://www.thelancet.com/journals/laneme/article/PIIS3050-5054(26)00005-0/fulltext?dgcid=hubspot_email_conferencealerts_gmi16&utm_campaign=conferencealerts&utm_medium=email&_hsenc=p2ANqtz-_w9lEQaNptxFCdrRsDnCz3SzqU1kOeUSWp1UkYZl803TSsBkQQ86GmLHxjAqqhMPNOMA5TjY-fxD8E1VPkL0SAR5d1ZQ&_hsmi=440685191&utm_content=440685191&utm_source=hs_email

High-grade gliomas show distinct biology across age and sex Proteogenomic analysis of brain tumors identified potential treatment targets and prognostic markers that differed across age, developmental stage, and sex. Written byBree Foster, PhD

High-grade gliomas show distinct biology across age and sex Proteogenomic analysis of brain tumors identified potential treatment targets and prognostic markers that differed across age, developmental stage, and sex. Written byBree Foster, PhD High-grade gliomas (HGGs) are highly aggressive primary brain tumors, with a 5-year survival rate below 10 percent. Recent genomic and epigenomic profiling studies have revealed key distinctions between adult HGG and pediatric HGG, suggesting they are biologically distinct diseases. Reflecting these differences, the 2021 WHO Classification of Tumors of the Central Nervous System made major changes to the classification of HGGs, separating pediatric and adult tumors into distinct categories. However, this leaves a less clearly defined group in between: adolescents and young adults (AYA), whose tumors can fall between the biological profiles of pediatric and adult disease. https://www.drugdiscoverynews.com/high-grade-gliomas-show-distinct-biology-across-age-and-sex-17544

Claude spots a mystery enzyme scientists overlooked Autonomous agents flagged a family of phage enzymes with CRISPR-like repeats, though what the system does remains unknown. Written byAndrea Corona

Claude spots a mystery enzyme scientists overlooked Autonomous agents flagged a family of phage enzymes with CRISPR-like repeats, though what the system does remains unknown. Written byAndrea Corona https://www.drugdiscoverynews.com/claude-spots-a-mystery-enzyme-scientists-overlooked-17549 AI has already changed how scientists predict protein structures and design new molecules. The next leap may be discovery itself, with agents that read raw biological data, spot what looks strange, and chase it down on their own. A new study offers an early glimpse of that future, and of what it could mean for the hunt for the next generation of therapeutic tools. Researchers at Anthropic reported that a team of autonomous large language model (LLM) agents surveyed reverse transcriptase (RT) genes across 1.9 billion metagenomic protein clusters and surfaced a new family of the enzymes, along with the repeat arrays that define it.

Ulefnersen slows disease progression in Phase 3 ALS trial A win for autoimmune disease, a set back for Alzheimer’s disease, an FDA backing for a multi-cancer blood test, and more led the news this week. Brought to you byDDN editorial team

Ulefnersen slows disease progression in Phase 3 ALS trial A win for autoimmune disease, a set back for Alzheimer’s disease, an FDA backing for a multi-cancer blood test, and more led the news this week. Brought to you byDDN editorial team Otsuka and Ionis Pharmaceuticals’ investigational RNA-targeted therapy ulefnersen has met the primary endpoint in a Phase 3 trial of FUS-associated amyotrophic lateral sclerosis (FUS-ALS), a rare and rapidly progressive form of the disease caused by mutations in the FUS gene. The FUSION study enrolled 89 patients, with 73 included in the primary analysis, and found that ulefnersen significantly slowed the combined measure of functional decline and survival at week 72 compared with placebo. The therapy also outperformed placebo on secondary measures, including a biomarker of neurodegeneration and time to death, permanent ventilation, rescue treatment, or withdrawal due to disease progression. Ulefnersen is designed to reduce the production of FUS protein by binding FUS pre-messenger RNA, including mutant forms that contribute to motor neuron degeneration. Otsuka plans to discuss the results with regulatory authorities and has launched an early-access program for eligible patients with genetically confirmed FUS-ALS who cannot participate in a clinical trial. There are currently no approved therapies specifically targeting the underlying genetic cause of FUS-ALS. – Bree Foster https://www.drugdiscoverynews.com/ulefnersen-slows-disease-progression-in-phase-3-als-trial-17546

Lessons From the Real World: Treating High Risk HR+/HER2- Breast Cancer According to the Latest Evidence Authors: Nadia Harbeck, MD, PhD; Stephen Johnston, MA, FRCP, PhD; Sara Tolaney, MD, PhD

https://www.medscape.org/viewarticle/lessons-real-world-treating-high-risk-hr-her2-breast-cancer-2026a1000bhd?page=1&src=mkmcmr_driv_stan_mscpedu_260924-OUS-HONC-lessons-real-world-treating-high-risk-hr-her2-breast-cancer-2026a1000bhd-cta&uac=148436CN

NIH launches new PubMed tool to strengthen research replication and reproducibility

NIH launches new PubMed tool to strengthen research replication and reproducibility The National Institutes of Health (NIH) today launched Linked Discoveries, an experimental tool designed to help scientists more easily see how an individual research finding relates to the larger body of biomedical evidence. From a citation in PubMed, users can use the Linked Discoveries tool to explore a “neighborhood” of publications closely related to that article, including replication studies. More than 29 million PubMed publications are represented in Linked Discoveries at launch. https://www.nih.gov/news-events/news-releases/nih-launches-new-pubmed-tool-strengthen-research-replication-reproducibility U.S. Department of Health and Human Services NATIONAL INSTITUTES OF HEALTH NIH News National Institutes of Health For Immediate Release: Thursday, September 24, 2026 CONTACT: NIH Office of Communications, NIH LAUNCHES NEW PUBMED TOOL TO STRENGTHEN RESEARCH REPLICATION AND REPRODUCIBILITY The National Institutes of Health (NIH) today launched Linked Discoveries, an experimental tool designed to help scientists more easily see how an individual research finding relates to the larger body of biomedical evidence. From a citation in PubMed, users can use the Linked Discoveries tool to explore a “neighborhood” of publications closely related to that article, including replication studies. More than 29 million PubMed publications are represented in Linked Discoveries at launch. Developed by NIH’s National Library of Medicine (NLM), which oversees PubMed, Linked Discoveries is an early product of NIH’s agency-wide initiative to strengthen replication and reproducibility in NIH-funded research. In particular, this tool seeks to address the difficulty of determining how a finding fits within the existing evidence. Relevant studies and other research information may be dispersed across different resources, and traditional approaches to finding scientific literature can favor already prominent or frequently cited work. Linked Discoveries begins to address that challenge by giving researchers a more connected view of publications related to a particular study. “With modern science racing forward at incredible speed, we face an ever-growing volume of biomedical literature but lack a way to understand how it relates to one another or contributes to the broader scientific context,” said NIH Director Jay Bhattacharya, M.D., Ph.D. “Finding the right information and related publications isn’t just about matching keywords, it’s about understanding how ideas connect, how findings build on one another, and how research evolves as new evidence emerges. Linked Discoveries was designed to support that level of exploration.” Using an AI-informed approach, Linked Discoveries identifies related publications and allows users to examine connections among them through graph and timeline views. Researchers can narrow a neighborhood by conditions, genes and chemicals and see information such as citation connections, reviews, retractions and NIH-funded publications. That context can help researchers determine what evidence should be considered as they interpret previous findings and plan subsequent research. Linked Discoveries does not judge the quality of a study or determine whether a finding has been successfully replicated. Instead, it organizes connections among publications and provides researchers with additional context to evaluate the evidence for themselves. “Rather than treating published literature as a static collection of facts, Linked Discoveries enables dynamic and discerning engagement,” said NLM Director Peter Embí, M.D. “By allowing users to explore the ‘neighborhood’ of related articles surrounding a publication, we can give researchers a broader view of the work that came before and after it, helping them explore context and identify questions that may warrant further investigation.” The September 24 launch is the first public version of Linked Discoveries. NLM will continue to test and refine the tool informed by user feedback — which can be submitted directly within the tool — to help inform future features and capabilities. The resource is designed to grow alongside NIH’s broader work to strengthen replication and reproducibility, while keeping scientific assessment in the hands of researchers. The National Library of Medicine (NLM) is a leader in research in biomedical informatics and data science and the world’s largest biomedical library. NLM conducts and supports research in methods for recording, storing, retrieving, preserving, and communicating health information. NLM creates resources and tools that are used billions of times each year by millions of people to access and analyze molecular biology, biotechnology, toxicology, environmental health, and health services information. Additional information is available at https://www.nlm.nih.gov. About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov. NIH...Turning Discovery into Health -- Registered, U.S. Patent and Trademark Office --- ### This NIH News Release is available online at: