viernes, 21 de agosto de 2026
One of the most consequential years for cancer treatment, ever It’s not hyperbole when you look at the achievements so far By Adam FeuersteinAug. 20, 2026 Senior Writer, Biotech
https://www.statnews.com/2026/08/20/cancer-vaccine-merck-moderna-biotech-observations/
By Adam FeuersteinAug. 20, 2026
Senior Writer, Biotech
Lil Nas X’s bipolar diagnosis and the paradox of treatment My patients fear that mental health treatment will make them ordinary
By Rachel DocekalAug. 21, 2026
Docekal is CEO of the Hanley Foundation, an organization focused on addiction prevention, treatment, and recovery services.
https://www.statnews.com/2026/08/21/lil-nas-xs-bipolar-diagnosis-treatment-medication-achievement/
A cure for HIV would be a triumph. But it shouldn’t harm survivors Without safeguards, scientific progress could trigger a benefits cliff
https://www.statnews.com/2026/08/21/hiv-cure-survivors-harm-benefits-research-ryan-white-hiv-aids-program/
By Ali Ahmed, Jeff Taylor, and Jeff BerryAug. 21, 2026
Ahmed is a postdoc researcher at Perelman School of Medicine, University of Pennsylvania. Taylor is director of the HIV+Aging Research Project-Palm Springs and a long-term survivor of HIV. Berry is the executive director of The Reunion Project, the national alliance of long-term survivors of HIV.
Focal therapy for localized prostate cancer has divided physicians. Will new data change minds? Recent study found 10-year survival was comparable to surgery or radiation, with fewer side effects
https://www.statnews.com/2026/08/21/focal-therapy-prostate-cancer-still-experimental-or-viable-alternative/?utm_campaign=morning_rounds&utm_medium=email&_hsenc=p2ANqtz-991g3erGfAD2icG4rzlBwFVgVi_c46DHNE0NrFq7iVyqptCtI1a4dll-OvrqBIFTnhOwn6zwlH2Vj0pG8-rrHqBJHrIw&_hsmi=434260558&utm_content=434260558&utm_source=hs_email
By Annalisa MerelliAug. 21, 2026
Contributing Writer
Rome Criteria for Irritable Bowel Syndrome: When Is Further Testing Needed? Teresa Santos; Ilana Polistchuck
https://www.medscape.com/viewarticle/rome-criteria-irritable-bowel-syndrome-when-further-testing-2026a1000sxo?ecd=wnl_edit_tpal_etid8617923&uac=148436CN&impID=8617923
Does knowing the diagnostic criteria necessarily lead to sound clinical decision-making? Not always. A recent study published in Neurogastroenterology & Motility evaluated medical students from 14 Latin American countries, including Brazil, and found that although most were familiar with the Rome Criteria for disorders of gut-brain interaction (DGBI), many still relied on a diagnosis of exclusion rather than a positive diagnosis. Researchers at the University of Miami in Miami found that this approach persisted even among students who had received formal training in DGBI.
New US Dietary Guidelines Get Big Picture Right but Misinterpret Critical Components for Liver Health Winston Dunn, MD; Ashwani K. Singal, MD, MS
https://www.medscape.com/viewarticle/new-us-dietary-guidelines-get-big-picture-right-misinterpret-2026a1000s6d?ecd=wnl_edit_tpal_etid8617923&uac=148436CN&impID=8617923
The Dietary Guidelines for Americans (DGA) 2025-2030 highlight several simple recommendations that are good for population health. For example, they focus on consuming vegetables, fruits, whole grains, protein, and dairy, and deemphasize highly processed foods, refined carbohydrates, sugary drinks, added sugars, and excess sodium. An eating pattern centered on unprocessed, familiar foods instead of packaged calories is exactly what clinicians advise for metabolic dysfunction-associated steatotic liver disease (MASLD).
Viral Spicy Food Challenges: Have They Become Too Risky? Stéphanie Lavaud
https://www.medscape.com/viewarticle/viral-spicy-food-challenges-have-they-become-too-risky-2026a1000ss3?ecd=WNL_trdalrt_pos1_ous_260821_etid8618434&uac=148436CN&impID=8618434
In recent years, online challenges involving extremely spicy foods and hot chili peppers have gained popularity. Although most individuals experience an intense burning sensation, some may develop gastrointestinal symptoms and severe allergic reactions in rare cases. The French Agency for Food, Environmental and Occupational Health & Safety (ANSES) has issued a warning, particularly for adolescents.
NEW PODCAST: The CheckRare Brief + +
NEW PODCAST: The CheckRare Brief
Episode 2: FDA Approves New Narcolepsy Treatment
On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients with SYNGAP1-related disorders, and BioMarin’s termination for their ENPP1 deficiency program following mixed results from their phase 3 clinical trial.
https://checkrare.com/fda-approves-new-narcolepsy-treatment-the-checkrare-brief/
Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia
https://checkrare.com/fda-advisory-boards-concerns-about-new-duchenne-drug-brain-fog-in-immune-thrombocytopenia/
The CheckRare Brief delivers a concise summary of the week’s most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.
FDA Approves Multiple Myeloma Treatment, Denies Neuroendocrine Tumors Treatment by CheckRare Staff| Published on: Aug 20, 2026
Patient Perspective: Diagnostic Journey With IgA Nephropathy by Madaline Spencer| Published on: Aug 21, 2026
Patient Perspective: Diagnostic Journey With IgA Nephropathy
by Madaline Spencer| Published on: Aug 21, 2026
Byron Wade, a patient with IgA nephropathy (IgAN), discusses his personal diagnostic journey and a recent study looking at other patients’ experiences.
https://checkrare.com/patient-perspective-diagnostic-journey-with-iga-nephropathy/
Overcomplicated GLP-1 Guidelines May Discourage Prescribing Yoni Freedhoff, MD
https://www.medscape.com/viewarticle/overcomplicated-glp-1-guidelines-may-discourage-prescribing-2026a1000qq0?ecd=mkm_ret_260821_mscpmrk-OUS_ICYMI_etid8612371&uac=148436CN&impID=8612371
The Lancet Diabetes & Endocrinology recently published a consensus piece on using GLP-1 medications for obesity in Europe. The paper, "Nutritional, Functional, and Psychological Considerations for Incretin-Based Therapies in Adults—an EASO, EFAD, and ECPO Consensus Statement", aimed to provide guidance on best practices for clinicians prescribing obesity medications — but one could argue, it's more likely to lead clinicians to feel ill equipped than empowered.
jueves, 20 de agosto de 2026
The growing role of multi-omics within drug discovery Harnessing multi-omics and AI, researchers are transforming drug discovery by revealing disease mechanisms, pinpointing therapeutic targets, and speeding up development. Written byBree Foster, PhD
The growing role of multi-omics within drug discovery
Harnessing multi-omics and AI, researchers are transforming drug discovery by revealing disease mechanisms, pinpointing therapeutic targets, and speeding up development.
Written byBree Foster, PhD
https://www.drugdiscoverynews.com/the-growing-role-of-multi-omics-within-drug-discovery-17097?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz--eZkE2RFdsdMb8DN-kmWB7uVa8nUXSe_FENTgeLw6eyAzhg2Iv9oKOCJcXyI-8gDfb-AfL_hKpZGbeNQRdhcBe6WJn0A&_hsmi=434032591&utm_content=434032591&utm_source=hs_email
Drug discovery has long been a high-risk, high-reward endeavor. Developing a new therapeutic from initial concept to market approval can take over a decade and cost more than $2 billion on average. Despite these staggering investments, the failure rate remains high, with many drugs faltering in late-stage clinical trials due to unforeseen toxicity, lack of efficacy, or poor patient stratification. Traditional approaches often focus on targeting a single molecule or pathway, an approach increasingly recognized as insufficient for tackling complex diseases such as cancer, neurodegeneration, and autoimmune disorders.
Can better training data fix AI antibody design?
The field has invested heavily in building better models for antibody discovery. The structural interaction data those models are trained on has not kept pace — and that shortfall is now a defining constraint on what AI can reliably do.
Written byAndrea Corona
https://www.drugdiscoverynews.com/can-better-training-data-fix-ai-antibody-design-17211?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-9IHi0YXjvamui5CmnXpL0g-NVgV9akzvA8Ls0j41AjlkP-O-L47wYGyJc68CPWAD6TcLZFCq8ES_RyJ048aEny6RQKcg&_hsmi=434032591&utm_content=434032591&utm_source=hs_email
The last several years of progress in protein artificial intelligence (AI) have been undeniably impactful. AlphaFold's demonstration that protein folding could be predicted with near-experimental accuracy reset expectations across structural biology, and the models that followed, for protein design, interaction prediction, and sequence generation, have moved antibody discovery into a new computational era.
AI-based analysis offers new roadmap for precision oncology The new approach combined digital pathology with spatial proteomics to uncover the secrets of metastasis and personalized treatment options. Written byAllison Whitten, PhD
AI-based analysis offers new roadmap for precision oncology
The new approach combined digital pathology with spatial proteomics to uncover the secrets of metastasis and personalized treatment options.
Written byAllison Whitten, PhD
https://www.drugdiscoverynews.com/ai-based-analysis-offers-new-roadmap-for-precision-oncology-17460
How and why a tumor metastasizes — when a seemingly similar tumor doesn’t — remains one of the biggest questions in cancer research.
New research from György Marko-Varga’s lab at Lund University with Istvan Nemeth at the Szeged Clinical hospital, and Peter Horvath’s team at HUN-REN Biological Research Centre suggests a new way to chip away at this question with the help of AI combined with spatial proteomics to reveal the single-cell dynamics of different populations of cancer cells within the same tumor. Their approach offers a promising step up over traditional molecular analyses that view the entire tissue sample at once and miss the functional differences in how different cell types behave.
As Healthy As Possible A Non-Profit Focused On POLR2A
https://ashealthyaspossible.net/
As Healthy as Possible
As Healthy as Possible, which works to make research benefiting people with a POLR2A gene mutation more accessible and visible, has released a new newsletter. It shares one family’s journey to diagnosis and highlights the POLR2A Patient Registry, which explores how different mutations relate to symptoms and disease severity. People with a POLR2A genetic testing report can contribute by completing the available surveys.
https://download2.eurordis.org/As_Healthy_As_Possible_Newsletter_June.pdf
What are Lysosomal Storage Diseases? + + + +
Following a general animation on Lysosomal Storage Diseases, the Platt Lab, in collaboration with patient associations, has created new animations on Niemann-Pick Diseases and GM2 gangliosidoses. Available in several languages, they explain each condition, diagnosis and treatment options, and signpost sources of patient and mental health support. Watch the animation on Niemann-Pick Diseases and the animation on GM2 gangliosidoses.
https://plattlab.nsms.ox.ac.uk/
What are Niemann-Pick Diseases?
What are the GM2 gangliosidoses: Tay-Sachs and Sandhoff disease?
ERDERA has launched its Clinical Trial Call (ECTC) to support multinational, GCP‑compliant early‑phase interventional clinical trials in rare diseases.
https://erdera.org/call/ctc2026/
ERDERA (the European Rare Diseases Research Alliance) has launched a Clinical Trial Call to support multinational, GCP-compliant Phase I, Phase I/II and Phase II interventional trials in rare diseases. Funding aims to generate robust, regulatory-relevant clinical evidence, with priority given to paediatric and rapidly progressive rare diseases, and those with no approved treatment options. Applications are open to research institutes, clinical centres, NGOs, patient advocacy organisations and SMEs. The deadline is 10 September.
Raising awareness on managing competing interests in a multi-stakeholder environment: Guidance to patients and engaging stakeholders + + +
https://imi-paradigm.eu/PEtoolbox/conflict-of-interest/
Conflict of Interest
Interested in learning how to manage competing interests in a multi-stakeholder environment? The EURORDIS Open Academy is offering a course providing guidance to patients on best practices for multi-stakeholder interactions. Based on a guide from PARADIGM, this course explores how to avoid and minimise conflict of interest by suggesting risk mitigation strategies.
https://openacademy.eurordis.org/
Conflict of interest in patient engagement
https://openacademy.eurordis.org/courses/conflict-of-interest-in-patient-engagement/
The JARDIN Hackathon to Seek Solutions to Overcome Technical Barriers in Health Data Exchange: From the Point of Care to European Registry Networks
https://datascience.codata.org/articles/10.5334/dsj-2026-029
This article describes the methods and outcomes of a JARDIN hackathon exploring technical barriers to secure health data exchange between healthcare providers, national registries and ERNs. The authors, including Veronica Popa, our Digital Patient Engagement Manager, present potential solutions to improve the exchange and interoperability of rare disease data.
Medical device development and innovation for rare and pediatric populations: a global landscape overview
https://link.springer.com/article/10.1186/s13023-026-04351-0
This review by the IRDiRC Working Group on Medical Technologies for Rare Diseases examines the global regulatory landscape, current initiatives and patient engagement in orphan device development. It identifies remaining gaps and calls for greater regulatory harmonisation and stronger collaboration among stakeholders.
Support Structure for Meaningful Patient Partnership in the European Reference Networks (ERNs): A Guide for ERN Coordinating Teams
https://www.eurordis.org/publications/support-structures-for-meaningful-patient-partnership-in-the-european-reference-networks/
EURORDIS has published a guide for ERN coordinating teams on embedding meaningful patient partnership across the ERNs. It explores the conditions, approaches and competencies needed to support sustained collaboration between patient representatives, health professionals and ERN project managers.
How a Swiss lab is finding new cures in old drugs
https://www.swissinfo.ch/eng/new-treatments/how-a-swiss-lab-is-finding-new-cures-in-old-drugs/91640346
Last month, Swissinfo published an article exploring the potential of drug repurposing to accelerate treatments for rare diseases, featuring insights from Claudia Fuchs, our Senior Project Manager. Claudia highlighted the barriers that can prevent promising research from reaching patients, particularly where commercial incentives are lacking.
SAVE THE DATE – Friday 13 November – Dublin, Ireland a rare disease themed Associated Event of Ireland’s Presidency of the Council of the European Union
https://rdi.ie/eu-presidency/
More than 30 million people across Europe live with a rare disease. This event explores how research and innovation can become the standard of care for all rare diseases — building momentum for a coordinated EU Action Plan, examining the Biotech Act’s role in strengthening Europe’s innovation base and competitiveness, and demonstrating what happens when patients are true partners.
We will demonstrate that with the right policy and with patients as partners in research and innovation, discoveries move more quickly from bench to bedside, innovation becomes more effective, and better outcomes become possible for people living with rare diseases. Europe can regain leadership in rare disease innovation.
10 Minutes With Rhiannon Walls + + +
10 Minutes With...
Catch up on the latest episode of 10 Minutes With, where host Rhiannon Walls is joined by Sarah Baker, Chief Executive of the Cambridge Rare Disease Network. In this discussion, Sarah outlines the story behind the Rare Patient Passport and how it was developed in response to the needs of the rare disease community. Together, they explore how this innovative tool is helping to address fragmented medical information, supporting healthcare professionals and families as they navigate diagnosis and treatment.
https://www.eurordis.org/10-minutes-with-rhiannon-walls/
Our vision is a world where everyone
affected by a rare condition feels
connected, empowered, and supported
https://www.camraredisease.org/
Webinar Series: Kickstart your advocacy August 2026
https://www.eurordis.org/webinar-series-kickstart-your-advocacy/
Kickstart Your Advocacy
A new webinar series is launching in September, designed to help young people in the rare disease community strengthen their advocacy and create meaningful change. Providing practical tools, knowledge and skills, the series will explore four key areas: communication and storytelling, leadership, European advocacy, and mental wellbeing and resilience.
Joint Clinical Assessment report on tovorafenib (Ojemda) + +
https://health.ec.europa.eu/publications/joint-clinical-assessment-report-tovorafenib-ojemda_en
HTA Regulation
Last month, EURORDIS released an evaluation of the first EU Joint Clinical Assessment (JCA) report on tovorafenib, published by the European Commission under the Health Technology Assessment (HTA) Regulation. By developing a scoring grid and drawing on input from an evaluation group of patient advocates, EURORDIS identified areas for improvement in future JCA reports and provided recommendations on structure, contributors and scientific content..
https://www.eurordis.org/eurordis-members-score-the-first-eu-joint-clinical-assessment-report/
Seven countries vote against Commission's health programme Ten member states abstained, and ten supported the EU4Health programme for 2026
https://www.euractiv.com/news/seven-countries-vote-against-commissions-health-programme/
On 22 July, seven EU Member States voted against the European Commission’s proposed 2026 EU4Health Work Programme over the absence of operating grants for health NGOs. As reported by Euractiv, EURORDIS welcomed the intervention as support for a “strong, independent and adequately funded” health civil society. On 31 July, a blocking minority again withheld approval after the Commission proposed €1.3 million in operating grants, still far below previous funding.
https://www.linkedin.com/pulse/july-2026-eurordis-iufve/
A Lottery at Birth: Why the EU needs a shared approach to newborn screening August 2026 + + +
https://www.eurordis.org/lottery-at-birth-europe-needs-shared-newborn-screening-approach/
Our latest article explores how fragmented newborn screening (NBS) programmes across Europe can leave children with very different chances of receiving an early diagnosis and timely care, depending on where they are born.
The article builds on the joint Position Statement we published in June, calling for stronger EU-level collaboration to help Member States strengthen their NBS programmes and make better use of shared evidence and expertise.
https://www.eurordis.org/publications/position-statement-eu-level-collaboration-on-newborn-screening/
Rare disease organisations can still endorse the Position Statement and support our call for a multi-stakeholder European Newborn Screening Group until 30 September.
https://form.jotform.com/eurordisforms/endorse-eurordis-nbs-statement
Regenerative Medicine Advanced Therapy Designation: US Food and Drug Administration FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia
https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation?utm_medium=email&utm_source=govdelivery
The U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) to reduce daily cornstarch intake as an adjunct to nutritional management in adults and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa), a rare, inherited genetic disorder caused by a deficiency of the enzyme glucose-6-phosphatase, which prevents the body from properly breaking down stored glycogen into glucose. Genglycos is the first approved treatment for this condition.
The accelerated approval pathway allows the FDA to approve certain drugs or biologics intended to treat a serious or life-threatening disease or condition if the agency determines the treatment has an effect on a surrogate endpoint or intermediate clinical endpoint that is reasonably likely to predict clinical benefit.
“Patients with GSDIa face possible life-threatening complications and have limited treatment options that include primarily life-long, strict dietary management,” said Karim Mikhail, B. Pharm., M.S., acting director of the Center for Biologics Evaluation and Research (CBER). “Today’s approval is a great milestone in using a gene therapy to treat this disease and improve the quality of life for people with this condition.”
The agency based this accelerated approval on clinical trial data showing Genglycos helps reduce daily cornstarch intake, when taken with an appropriate diet. Reducing cornstarch intake is the surrogate endpoint, and the manufacturer must complete additional clinical trials to confirm Genglycos’s effectiveness.
GSDIa is caused by a genetic mutation that results in a missing enzyme called glucose-6-phosphatase (G6PC). This enzyme normally releases free glucose (a simple sugar and main source of energy for the body) from the liver and kidneys into the bloodstream, helping to maintain stable blood sugar levels during fasting and between meals. This enzyme deficiency causes the blood sugar to drop dangerously low whenever a person goes too long without eating. The condition can lead to long-term metabolic complications that can impair normal function in certain organs and tissues. GSDIa is typically managed through careful medical monitoring, frequent meals, avoidance of certain foods containing simple sugars, and daily, strict, around-the-clock dietary supplementation of uncooked or specially formulated cornstarch (slow-digesting complex carbohydrate) to prevent hypoglycemia (abnormally low blood sugar levels).
Genglycos is a one-time AAV8 based gene therapy designed to deliver a functional G6PC gene to the liver, aiming to restore the deficient enzyme required to release stored glucose and ensure stable blood sugar levels when a person is fasting.
“Genglycos offers these patients and their families a one-time therapy that targets the root cause of the disease,” said Megha Kaushal, M.D., MSc., acting deputy director of the CBER Office of Therapeutic Products. “This accelerated approval reflects our confidence in the clinical evidence to date and our commitment to bringing innovative treatments to patients with rare genetic diseases while we continue to gather data to confirm long-term benefit.”
The effectiveness of Genglycos was evaluated in a randomized, double-blind, placebo-controlled study in patients with GSDIa followed over 48 weeks after dosing. Patients treated with Genglycos demonstrated a statistically significant mean reduction from baseline in daily cornstarch intake of 31% compared to placebo, the study’s primary endpoint. A mean reduction from baseline of one cornstarch dose per day was seen in the Genglycos group compared to placebo, the study’s secondary endpoint. Genglycos-treated patients experienced a numerical (mean 3%) increase in the percentage of glucose values in the hypoglycemic range (< 70 mg/dL) compared to placebo.
Across two clinical studies of Genglycos including the randomized clinical study, serious adverse reactions reported in Genglycos-treated patients included anaphylaxis, adrenal insufficiency (a condition where the adrenal glands do not produce enough stress hormones), high lactate levels, and hypoglycemia. The most commonly reported adverse reactions included increased transaminases (elevation of liver enzymes), nausea, headache, constipation, and hyperglycemia. Genglycos-treated patients had a higher rate of hypertriglyceridemia (high blood triglyceride levels), a metabolic marker of GSDIa, compared to placebo-treated patients (29% vs 8%). The prescribing information contains warnings about the risks of anaphylaxis, liver toxicity, adrenal insufficiency, and risk of tumorigenicity (development of tumors). Genglycos should not be used during pregnancy.
The application for Genglycos received a Rare Pediatric Disease Priority Review Voucher. The FDA granted Genglycos regenerative medicine advanced therapy (RMAT) and Fast Track designations.
Rare Pediatric Disease Designation and Priority Review Voucher Programs
Information for product sponsors
https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs?utm_medium=email&utm_source=govdelivery
An Enduring Mission in a New Era: The FDA Public Health Pillars: FDA Authorizes First-Of-Its-Kind Robotic Blood Draw Device
https://www.fda.gov/news-events/fda-voices/enduring-mission-new-era-fda-public-health-pillars?utm_medium=email&utm_source=govdelivery
The U.S. Food and Drug Administration today authorized the Aletta, the first standalone robotic device that can draw blood from a patient’s arm without hands-on operator intervention. The device is authorized for use in adults in outpatient settings and must be operated under the oversight of a supervisor trained in phlebotomy. One phlebotomist can oversee up to three Aletta devices at the same time, which may help address the current phlebotomist shortage in the U.S.
“This authorization reflects the FDA’s commitment to advancing innovative medical devices that help meet a critical public health need while maintaining the safety and effectiveness patients deserve,” said Michelle Tarver, M.D., Ph.D., Director of the FDA’s Center for Devices and Radiological Health. “Blood draws are one of the most commonly performed medical procedures in the United States, yet patients may face delays due to a growing shortage of trained phlebotomists.”
The Aletta autonomously handles each step of the blood draw, while a trained phlebotomist initiates each session and remains available to respond to any issues throughout. The device guides the patient to position their arm, after which the patient or supervisor presses a button to start the blood draw. The Aletta then uses near-infrared light and Doppler ultrasound to locate a suitable vein and tell it apart from arteries. If no appropriate vein is found, the device will not attempt the procedure.
Once a vein is identified, the Aletta proceeds through the remaining steps on its own: applying a tourniquet, preparing the skin, inserting and disposing of the needle, changing collection tubes and placing a bandage. The supervising phlebotomist confirms that collection tubes are filled in the correct order and verifies that all tubes are adequately full following the procedure.
The Aletta is designed with multiple layers of patient safety in mind. The device continuously applies disinfectant to the skin during the ultrasound scan. The device is also cleaned by a trained professional between patients. If a patient moves too much during the procedure, the needle automatically detaches and the draw stops; additional onboard sensors can pause the procedure and alert the supervisor if other unsafe conditions are detected.
About the Authorization
This authorization advances the FDA’s Public Health Pillars of Innovation and Global Leadership, Increasing Access to Affordable Medicines and Medical Products, and Preventing Chronic Disease and Promoting Wellness in America by bringing a transformative technology to patients who increasingly face delays in accessing a critical diagnostic service.
The FDA’s authorization is based on clinical data demonstrating that the Aletta achieves successful blood draw rates comparable to or better than trained human phlebotomists when it proceeds with a stick. This was demonstrated across a broad range of patients, including those with varying health statuses, those who self-reported having difficult vein access and those with varying skin tones. Device-related adverse events were uncommon and mild.
The FDA granted marketing authorization to Vitestro for the Aletta through the De Novo pathway, a regulatory pathway for low- to moderate-risk devices of a new type. Along with this De Novo authorization, the FDA is establishing special controls that define requirements including those related to labeling, performance testing and clinical testing. When met, the special controls, in combination with general controls, provide a reasonable assurance of safety and effectiveness.
FDA Approves Second Treatment for Fibrodysplasia Ossificans Progressiva
https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-second-treatment-fibrodysplasia-ossificans-progressiva?utm_medium=email&utm_source=govdelivery
The U.S. Food and Drug Administration has approved Pasatru (garetosmab-grts) to reduce new heterotopic ossification and clinician-assessed disease flare-ups in adults with fibrodysplasia ossificans progressiva (FOP).
FOP is a very rare genetic disease in which connective tissues — including muscle, tendons, and ligaments — progressively transform into bone outside the skeleton, causing severe disability and early death. Pasatru is a targeted therapy that works by blocking a faulty protein in the body. In this disease, that protein sends abnormal signals that cause soft tissue to turn into bone. By disabling this protein, Pasatru stops those signals at their source, helping to prevent further abnormal bone growth.
Pasatru is administered as an intravenous infusion once every four weeks. It received Breakthrough Therapy, Fast Track, Orphan Drug, and Priority Review designations. The approval was granted to Regeneron Pharmaceuticals, Inc.
miércoles, 19 de agosto de 2026
GLP-1 Receptor Agonists in Alzheimer’s Disease: Emerging Off-Label Neuroprotective Potential
https://www.news-medical.net/health/GLP-1-receptor-agonists-in-Alzheimere28099s-disease-Emerging-off-label-neuroprotective-potential.aspx
From reduced amyloid and neuroinflammation in experimental models to mixed cognitive outcomes in clinical trials, emerging evidence is testing whether GLP-1 receptor agonists can move beyond metabolic therapy to become meaningful treatments for Alzheimer’s disease.
GLP-1 Receptor Agonists in Migraine and Intracranial Hypertension: Emerging Off-Label Therapeutic Potential
https://www.news-medical.net/health/GLP-1-Receptor-Agonists-in-Migraine-and-Intracranial-Hypertension-Emerging-Off-Label-Therapeutic-Potential.aspx
GLP-1 receptor agonists are emerging as promising investigational therapies for idiopathic intracranial hypertension, with evidence suggesting reductions in intracranial pressure, headache burden, papilledema, and visual complications. Their potential role in primary migraine remains far less certain and requires dedicated randomized clinical trials.
What 3,600 adults with diabetes revealed about lifestyle and heart health
https://www.news-medical.net/news/20260819/What-3600-adults-with-diabetes-revealed-about-lifestyle-and-heart-health.aspx
A UK Biobank analysis followed more than 3,600 adults with type 2 diabetes to examine how diet, sleep, physical activity, and social habits relate to major cardiovascular events and survival.
In a recent study published in the journal eClinicalMedicine, researchers investigated associations between adherence to a Mediterranean lifestyle and the risk of major adverse cardiovascular events (MACE) and mortality in adults with type 2 diabetes (T2D).
Beyond the boardroom: how the Lions’ Den is redefining patient-industry dialogue at the International PBC Summit 2026 Written by Becky Pender, senior associate, RARE Revolution Magazine
https://rarerevolutionmagazine.com/beyond-the-boardroom-how-the-lions-den-is-redefining-patient-industry-dialogue-at-the-international-pbc-summit-2026/
The International PBC Summit has long been heralded as a crucial meeting point for all stakeholders invested in primary biliary cholangitis (PBC). Yet, year after year, it is the distinctive and often challenging Lions’ Den session that captures the greatest attention and drives the most profound outcomes. Taking place on day three of the summit, the Lions’ Den is not a passive forum or a polished presentation, but a direct, structured dialogue specifically designed to confront a central challenge in modern healthcare: the persistent lack of transparent and equal access for patients and advocates to the pharmaceutical companies developing their treatments.
The Summit saw approximately 20 patient advocacy groups (PAGs) attend, with four pharmaceutical companies participating in the session. This one-to-many relationship creates a uniquely controlled and transparent environment for engagement. The structure itself is simple yet highly effective: a company presents its work, insights or pipeline, followed immediately by a dedicated Q&A session where PAGs lead the questioning on topics ranging from new treatments and clinical trial design to the often-thorny issue of access to therapies. The rotation ensures every participating company is engaged one at a time, guaranteeing all PAGs participate in each interaction, thereby ensuring equity in access to knowledge.
The anatomy of trust and accountability
The core value of the Lions’ Den format lies in its capacity to create a rare space where PAGs can engage with industry as equal stakeholders. This standardisation is crucial for levelling the playing field. By ensuring that every company receives the same amount of time, the same format and is subjected to the same type of questioning, the mechanism reduces the inherent power imbalance that frequently exists between large, well-resourced pharmaceutical companies and smaller patient organisations with limited experience in high-stakes negotiations.
In this environment, PAGs are freed from the necessity to ‘perform diplomacy’ constantly. Instead, the format encourages genuine openness and accountability while maintaining respect. As one patient advocate described, this structure provides a necessary safety net:
“For smaller organisations especially, standardisation creates safety. It gives everyone the same opportunity to participate and ensures that the patient perspective is not overshadowed by influence, size or funding capacity.” – patient advocate
The discussions that emerge are often far more direct and practical than those in traditional settings, covering difficult questions about access, timelines, affordability, communication and unmet needs, which might otherwise be glossed over in overly polished or general meetings.
“The core value is creating a rare space where patient advocacy groups can speak openly and directly with industry as equal stakeholders. The format fosters honesty and accountability.” – PAG member
A single viral platform shows promise across infectious disease and cancer ReiThera’s gorilla-derived adenovirus vector is helping advance vaccines and therapies for COVID-19, HIV, Ebola, and even cancer. Written byBree Foster, PhD
https://www.drugdiscoverynews.com/a-single-viral-platform-shows-promise-across-infectious-disease-and-cancer-17103?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-9AVNke2GsjfUAH4klAHUbDwd9KUHvUb7XOLqpTqqD969vkKpbA_anyAowbUKoDN2KhFojXwlgJo1VRt3JP_tvUWDypmA&_hsmi=433938578&utm_content=433938578&utm_source=hs_email
At Advanced Therapies 2026, DDN spoke with Claudio Panzarella, Head of Business Development, and Angelo Raggioli, Head of Technology Development, at ReiThera, about how the company’s early COVID-19 vaccine efforts shaped its current scientific programs, including the gorilla adenovirus (GRAd) platform, ongoing HIV vaccine trials, and the potential for oncology therapeutics.
Triple drug therapy could clear HIV in infected newborns Scientists showed that a trio of medications successfully cleared the virus in a rhesus macaque infant model. Clinical testing in human infants could move quickly. Written byAllison Whitten, PhD
https://www.drugdiscoverynews.com/triple-drug-therapy-could-clear-hiv-in-infected-newborns-17446
In 2013, a baby girl known as the “Mississippi baby” made headlines when it was announced that she was cured of HIV. She had received antiretroviral therapy (ART) starting just 30 hours of birth up until 18 months, and had no detectable levels of the virus over two years later. But then, when she was almost four years old, her blood revealed the HIV had returned, and she had to start ART once again.
Endocrine Disorders
Endocrine Disorders
According to the NIH, there are more than 175 rare diseases that affect the endocrine system. In this section we highlight many of them and provide clinically relevant perspectives on these rare diseases.
https://checkrare.com/diseases/endocrine-disorders/
Congenital And Genetic Conditions
https://checkrare.com/diseases/congenital-and-genetic-conditions/
Congenital and genetic conditions can be inherited or caused by environmental factors and can vary from mild to severe. This section is focused on genetic and congenital disorders in children and adolescents.
Autoimmune / Autoinflammatory Disorders
https://checkrare.com/diseases/autoimmune-autoinflammatory-disorders/
Disease overviews, clinically relevant perspectives, and news about the most important research in rare autoimmune and auto-inflammatory disorders.
Importance of Patient Listening in The Development of Rare Disease Treatments by Madaline Spencer| Published on: Aug 19, 2026
Importance of Patient Listening in The Development of Rare Disease Treatments
by Madaline Spencer| Published on: Aug 19, 2026
Matt Trudeau, President of ITF Therapeutics, discusses the importance of patient listening in the development of Duvyzat (givinostat) for Duchenne muscular dystrophy (DMD).
https://checkrare.com/importance-of-patient-listening-in-the-development-of-rare-disease-treatments/
NOACs Linked to Slower Cognitive Decline in Comorbid AF and Alzheimer’s Disease Lizette Borreli
https://www.medscape.com/viewarticle/noacs-linked-slower-cognitive-decline-comorbid-af-and-2026a1000smf?ecd=WNL_trdalrt_pos1_ous_260819_etid8610903&uac=148436CN&impID=8610903
Non-vitamin K oral anticoagulant (NOAC) use was associated with modestly slower cognitive decline among older adults with comorbid atrial fibrillation (AF) and Alzheimer’s disease (AD).
martes, 18 de agosto de 2026
Type 1 Plasminogen Deficiency Overview by Madaline Spencer| Published on: Mar 13, 2026 + + +
Type 1 Plasminogen Deficiency Overview
by Madaline Spencer| Published on: Mar 13, 2026
Amy Shapiro, MD, a pediatric hematologist and the Medical Director and CEO of the Indiana Hemophilia and Thrombosis Center in Indianapolis, provides CheckRare an overview on type 1 plasminogen deficiency (PLGD-1).
https://checkrare.com/type-1-plasminogen-deficiency-overview/
Diagnosis and Management of Plasminogen Deficiency
by Madaline Spencer| Published on: May 11, 2026
Amol Sura, MD, Foster Center for Ocular Immunology, Dept of Ophthalmology, Duke University, Durham, North Carolina, discusses the diagnosis and management of plasminogen deficiency, a rare condition in which the eyes are first affected, but it manifests in mucous membranes throughout the body.
https://checkrare.com/diagnosis-and-management-of-plasminogen-deficiency/
Key Features of PLGD-1
by Madaline Spencer| Published on: May 19, 2026
Angela Zhu, MD, is an Ophthalmologist and Clinical Assistant Professor, Pediatric & Adult Cornea/Cataract/External Diseases, at Bascom Palmer Eye Institute in Miami. In this interview with CheckRare, Dr. Zhu describes some of the key features of plasminogen deficiency and why ophthalmologists are often the healthcare providers who begin the process toward diagnosis.
https://checkrare.com/diagnosis-and-management-of-plasminogen-deficiency-2/
A framework for mindfulness-based social-emotional learning in Vietnamese schools Phi-Bao Nguyen-Ngoc, Thien-Vu Giang, Van-Son Huynh Volume 3, Issue 3
https://www.academia.edu/journals/academia-mental-health-and-well-being/articles?source=journal-top-nav
Adolescent mental health has become an increasing concern in Vietnam and Southeast Asia, where schools are expected to play a stronger role in wellbeing promotion. Although social-emotional learning (SEL) and mindfulness-based approaches have both shown promise in promoting students’ psychological wellbeing, their integration in Vietnamese schools remains underdeveloped. This paper proposes a framework for mindfulness-based social-emotional learning (MBSEL) in Vietnamese schools. A narrative review was conducted through searches of Scopus, Web of Science, ERIC, PsycINFO, and Google Scholar, supplemented by reference-list screening, to identify English-language conceptual, empirical, and policy literature on SEL, mindfulness, adolescent development, school mental health, and culturally responsive education published up to June 2026. The literature was critically synthesized to examine conceptual connections, intervention evidence, and implications for Vietnam. The synthesis indicates that mindfulness and SEL are complementary. Existing MBSEL-related programs provide encouraging but uneven evidence, with stronger support for stress reduction, emotion regulation, and self-compassion than for interpersonal and school-climate outcomes. The proposed framework positions mindfulness as a facilitating mechanism supporting SEL competencies among students, teachers, and other school stakeholders, thereby contributing to safer, more caring, and respectful environments aligned with Vietnam’s Happy School initiatives. As a narrative review without formal quality or risk-of-bias appraisal, the synthesis may be affected by selection and language bias and does not support definitive causal conclusions. MBSEL should therefore be implemented through cultural adaptation, teacher preparation, whole-school support, and rigorous empirical evaluation.
https://www.academia.edu/2997-9196/3/3/10.20935/MHealthWellB8427
Can AI uncover a new path to non-opioid pain relief? As non-opioid pain therapies continue to face setbacks, Insilico is using AI to uncover new biology and therapeutic targets. Written byBree Foster, PhD
Can AI uncover a new path to non-opioid pain relief?
As non-opioid pain therapies continue to face setbacks, Insilico is using AI to uncover new biology and therapeutic targets.
Written byBree Foster, PhD
https://www.drugdiscoverynews.com/can-ai-uncover-a-new-path-to-non-opioid-pain-relief-17425
The search for effective non-opioid pain medicines has produced both breakthroughs and setbacks. While the approval of Vertex Pharmaceuticals’ non-opioid drug Journavx in 2025 offered a new option for acute pain, it didn’t see the immediate commercial success many analysts anticipated, with third-quarter sales falling $3 million short of expectations. This was quickly followed by Vertex then discontinuing a follow-up non-opioid candidate after disappointing clinical results. Meanwhile, Eli Lilly has shelved two non-opioid drug candidates.
Predicting psoriatic arthritis in new-onset psoriasis: development of multivariable prediction models from an inception cohort study Axel Svedbom, PhDa,b Send email to axel.svedbom@ki.se ∙ Lotus Mallbris, MDa,c ∙ Alen Zabotti, MDd ∙ Martin P Playford, PhDe ∙ Pernilla Nikamo, PhDa ∙ Nehal N Mehta, MDf ∙ et al.
Predicting psoriatic arthritis in new-onset psoriasis: development of multivariable prediction models from an inception cohort study
Axel Svedbom, PhDa,b Send email to axel.svedbom@ki.se ∙ Lotus Mallbris, MDa,c ∙ Alen Zabotti, MDd ∙ Martin P Playford, PhDe ∙ Pernilla Nikamo, PhDa ∙ Nehal N Mehta, MDf ∙ et al.
https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(26)00115-3/abstract?dgcid=raven_jbs_etoc_feature_lanrhe
Trends in autoimmune rheumatic disease diagnoses before and after the COVID-19 pandemic in England: a population-based cohort study using OpenSAFELY Mark D Russell, PhDa Send email to mark.russell@kcl.ac.uk ∙ Andrea Schaffer, PhDc ∙ Zijing Yang, PhDa ∙ Arti Mahto, PhDd ∙ Katie Bechman, PhDa ∙ Chris Wincup, PhDd ∙ et al.
Trends in autoimmune rheumatic disease diagnoses before and after the COVID-19 pandemic in England: a population-based cohort study using OpenSAFELY
Mark D Russell, PhDa Send email to mark.russell@kcl.ac.uk ∙ Andrea Schaffer, PhDc ∙ Zijing Yang, PhDa ∙ Arti Mahto, PhDd ∙ Katie Bechman, PhDa ∙ Chris Wincup, PhDd ∙ et al.
https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(26)00142-6/fulltext?dgcid=raven_jbs_etoc_feature_lanrhe
Translating progress into patient benefit in dermatomyositis The Lancet Rheumatology +...
Translating progress into patient benefit in dermatomyositis
The Lancet Rheumatology
https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(26)00256-0/fulltext?dgcid=raven_jbs_etoc_email
Sep 2026
Volume 8Number 9e675-e756
https://www.thelancet.com/journals/lanrhe/issue/vol8no9/PIIS2665-9913(26)X2008-2
lunes, 17 de agosto de 2026
Semaglutide Use and Risk for Depression Show No Clear Association
https://www.medscape.com/viewarticle/semaglutide-use-and-risk-depression-show-no-clear-2026a1000s4u?src=
The use of semaglutide was not associated with an increased risk for depression compared with placebo or other antidiabetic/antiobesity agents, but the evidence was limited and of very low certainty.
Cannabis Use May Increase Fall Risk in Adults With HIV
https://www.medscape.com/viewarticle/cannabis-use-may-increase-fall-risk-adults-hiv-2026a1000s50?src=
Cannabis use in the past month was associated with a higher risk for falls among adults living with HIV. However, after adjusting for other substance use, prescribed medications, and comorbidities, the association was no longer statistically significant.
Fact or Fiction: Multiple Sclerosis Diagnosis and Workup Reviewed by Christopher C. Luzzio, MD
https://reference.medscape.com/viewarticle/fact-or-fiction-multiple-sclerosis-diagnosis-and-workup-2026a1000nsr?src=&_gl=1*667rps*_gcl_aw*R0NMLjE3ODM4NzAxMzguQ2p3S0NBanc5c3pTQmhCTkVpd0FDNTdTcTJZUXZTZkkxc01raEZQZ1VLSWRQZkVUdGU4U212WEI0NGh3aEE5WmMtVDhTSVBEWlBzNmdSb0NiT1FRQXZEX0J3RQ..*_gcl_au*NjkzMDY5MjIxLjE3ODA2NzA2MDAuMTY4MzI1MDM3LjE3ODY5NzI2OTIuMTc4Njk3NDg1Mi4yMTI0OTM5ODg4LjE3ODY4OTIyNTQuMTc4Njk3NDg1Mg..
The 2024 revision of the McDonald criteria has substantially refined how clinicians diagnose multiple sclerosis (MS), placing greater emphasis on robust imaging and cerebrospinal fluid (CSF)-based evidence. The updated framework incorporates new MRI biomarkers, revised CSF parameters, and tailored guidance for specific patient subgroups, such as individuals with radiologically isolated syndrome (RIS) and older adults.
Mastectomy Preserves Ventriculoperitoneal Shunt in Woman
https://www.medscape.com/viewarticle/mastectomy-preserves-ventriculoperitoneal-shunt-woman-2026a1000s45?src=
Preserving a ventriculoperitoneal (VP) shunt during breast cancer surgery may be feasible when the tumor is close to the catheter and shunt dependence precludes removal. This case demonstrated that careful preoperative imaging, multidisciplinary planning, avoidance of energy devices near the catheter, and meticulous sharp dissection allowed the shunt to be preserved during mastectomy without damage or subsequent complications.
Fast Five Quiz: Psoriasis and Pregnancy Reviewed by Shari Lipner, MD, PhD
https://reference.medscape.com/viewarticle/psoriasis-and-pregnancy-2026a1000oia?src=&_gl=1*o84t4p*_gcl_aw*R0NMLjE3ODM4NzAxMzguQ2p3S0NBanc5c3pTQmhCTkVpd0FDNTdTcTJZUXZTZkkxc01raEZQZ1VLSWRQZkVUdGU4U212WEI0NGh3aEE5WmMtVDhTSVBEWlBzNmdSb0NiT1FRQXZEX0J3RQ..*_gcl_au*NjkzMDY5MjIxLjE3ODA2NzA2MDAuMTY4MzI1MDM3LjE3ODY5NzI2OTIuMTc4Njk3NDg1Mi4yMTI0OTM5ODg4LjE3ODY4OTIyNTQuMTc4Njk3NDg1Mg..
Because psoriasis can affect female patients during their reproductive years, pregnancy commonly overlaps with ongoing disease management. Clinical care during pregnancy focuses on balancing maternal disease control with fetal safety using evidence-based treatment decisions and including multidisciplinary input when needed.
Switching to Integrase Inhibitors Does Not Raise Long-Term Heart Disease Risk in People With HIV +++ +++ +
Integrase Inhibitors Safe for Heart in HIV Patients
Switching to Integrase Inhibitors Does Not Raise Long-Term Heart Disease Risk in People With HIV
Medscape Medical News 57 minutes ago
https://www.medscape.com/viewarticle/switching-integrase-inhibitors-does-not-raise-long-term-2026a1000s47?src=
One Third of Patients Achieve Clinically Meaningful Weight Loss With Dietitian-Led Primary Care Treatment
Early Dietitian Visits Linked to Successful Weight Loss
Medscape Medical News 1 hour ago
https://www.medscape.com/viewarticle/one-third-patients-achieve-clinically-meaningful-weight-loss-2026a1000s11?src=
Diabetes Drug Curbs Weight Gain in Older People With HIV
Diabetes Drug Curbs Weight Gain in Older People With HIV
Medscape News UK 2 hours ago
https://www.medscape.com/viewarticle/diabetes-drug-curbs-weight-gain-older-people-hiv-2026a1000rq9?src=
Obesity Class III Linked to Death Risk in Younger Adults
Medscape Medical News 55 minutes ago
https://www.medscape.com/viewarticle/obesity-class-iii-linked-increased-mortality-risk-younger-2026a1000s44?src=
Gram-Negative Bacteria Lead Pediatric Cancer BSIs
Medscape Medical News 1 hour ago
https://www.medscape.com/viewarticle/gram-negative-bacteria-dominate-bloodstream-infections-2026a1000s3s?src=
SLE Linked to Increased Risk for Venous Thromboembolism
Medscape News UK 2 hours ago
https://www.medscape.com/viewarticle/systemic-lupus-erythematosus-linked-increased-risk-venous-2026a1000ro9?src=
Cigars, Pipes Up Lung Cancer Risk, Raise Screening Needs
Medscape News UK 2 hours ago
https://www.medscape.com/viewarticle/cigar-and-pipe-smoking-increases-risk-lung-cancer-and-2026a1000rqr?src=
Vagal Nerve Stimulation for Rheumatoid Arthritis a Year After Approval: Where Do Things Stand? Richard Mark Kirkner
https://www.medscape.com/viewarticle/vagal-nerve-stimulation-rheumatoid-arthritis-year-after-2026a1000s3y?src=
Rheumatologist Ann Igoe, MD, has lived with debilitating rheumatoid arthritis (RA) since she was a 10-year-old girl in Ireland, but since she received a vagus nerve neuroimmune modulation implant in July that’s specifically indicated to treat RA, she said, “I actually felt like I won the lotto.”
RARE Revolution RARE Bite
https://editions.rarerevolutionmagazine.com/html5/reader/production/default.aspx?pubname=&edid=e0508b93-1cde-4785-b9ae-c36724bde55a&pnum=64
The International PBC Summit 2026, held from 20 to 23 April in Edinburgh, Scotland, by the PBC Foundation, represented an important patient‑centric healthcare collaboration. Far from a traditional conference, the Summit agenda was built upon a dynamic priority‑setting framework. This approach ensured that the work undertaken emerged from the collective wisdom of patients, clinical professionals, industry representatives, and advocates.
Topline Results from the MonumenTAL-6 Clinical Trial in Adults With Relapsed/Refractory Multiple Myeloma by Madaline Spencer| Published on: Aug 17, 2026
Topline Results from the MonumenTAL-6 Clinical Trial in Adults With Relapsed/Refractory Multiple Myeloma
by Madaline Spencer| Published on: Aug 17, 2026
Ajay K. Nooka, MD, MPH, FACP, Director of the Myeloma Program at the Emory University School of Medicine, discusses topline results from the MonumenTAL-6 clinical trial testing Tecvayli (teclistamab) in adults with relapsed/refractory multiple myeloma (RRMM).
https://checkrare.com/topline-results-from-the-monumental-6-clinical-trial-in-adults-with-relapsed-refractory-multiple-myeloma/
ICER: AstraZeneca hypertension drug is too expensive
https://icer.org/wp-content/uploads/2026/08/ICER_Hypertension_Draft-Report_For-Publication_081226.pdf?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-9uBnMRLalPLEhRK8bGLnNh6vHmUJPv1ozrbjVIHDxZY509xx0hGug-_oVpDXqOQh2H05n6PmGwxz2gezp79gbhfrjOcg&_hsmi=433538757&utm_content=433538757&utm_source=hs_email
ICER: AstraZeneca hypertension drug is too expensive
AstraZeneca’s newly approved hypertension drug Baxfendy is too expensive to meet traditional cost-effectiveness thresholds, according to a draft report from drug pricing watchdog ICER. The group assessed Baxfendy at its $10,800 annual list price. The group reached the same conclusion for Mineralys Therapeutics’ experimental rival lorundrostat.
ICER said the benefits of the two aldosterone synthase inhibitors are “promising but inconclusive” compared with cheap generic hypertension drugs.
AstraZeneca countered that Baxfendy, the first new class of hypertension drugs in more than two decades, produced clinically meaningful blood pressure reductions in a Phase 3 trial and could help millions of patients whose hypertension remains uncontrolled on multiple medications. AstraZeneca estimates that Baxfendy’s peak sales potential could be $5 billion after it acquired its original developer, CinCor Pharma, for $1.3 billion in 2023.
Europe pulls Tavneos over trial integrity concerns
https://www.ema.europa.eu/en/documents/referral/tavneos-article-20-procedure-scientific-conclusions_en.pdf?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-9a5A20SZYGd8M6Nb1b4vZ-d6og2aCZq4NTj6pOmmp0etKLMXS803RY8RHNly5-2SjlV8UU-VDYjFUI4D6TBJQkdxIVvg&_hsmi=433538757&utm_content=433538757&utm_source=hs_email
Europe pulls Tavneos over trial integrity concerns
European regulators last week formally revoked the marketing authorization for Tavneos, the vasculitis treatment developed by ChemoCentryx — a company acquired by Amgen for $4 billion.
The EMA concluded that the pivotal trial supporting its approval was unreliable, and that the sponsor personnel behind a Phase 3 study for the drug accessed unblinded efficacy data. Regulatory documents show that the EMA’s Committee for Medicinal Products for Human Use said the drug initially failed to demonstrate superiority at 52 weeks — before researchers re-adjudicated nine patients. This shifted the result from being statistically insignificant to significant, ultimately paving the path for approval, the agency said. Last month, NEJM retracted a paper outlining the Tavneos results.
https://www.reuters.com/legal/litigation/amgen-submits-new-data-challenge-fdas-proposal-withdraw-tavneos-2026-07-24/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-8vPlSbdrxD1TiXXCgdg4Yp48T04RiPySjC01HDXF0RBf3ufNTdE1nBymSTjz6cE7Gj3EJIRKMJRg0VkSAmJ557nEB4iA&_hsmi=433538757&utm_content=433538757&utm_source=hs_email
Regulators said the modifications weren’t adequately disclosed and amounted to a serious breach of good clinical practice. The agency also cited reports of fatal liver injury and vanishing bile duct syndrome, concluding that Tavneos’ benefits could no longer be shown to outweigh its risks.
Amgen disputes the decision. It’s making a similar case to the FDA after the agency asked the company in January to voluntarily withdraw Tavneos from the U.S. market. A few weeks ago, Amgen submitted real-world evidence and an independent reanalysis of the trial to regulators.
https://www.appliedclinicaltrialsonline.com/view/nejm-retracts-tavneos-trial-fda-ema-amgen-drug-market?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-9liArKFfWvFTUPZX0Jl2OcDna6h08jkGnx6JxuKmsl-5IXyPJWjSLaaUXwCeV7Sx63HTGMgQBOrmR1-fFLAtiQO58Y-Q&_hsmi=433538757&utm_content=433538757&utm_source=hs_email
Akeso and Summit’s ivonescimab extends survival in squamous cell lung cancer ‘Two drugs in one’ candidate, developed in China, is a highlight at ASCO By Matthew HerperMay 31, 2026 Senior Writer, Medicine, Editorial Director of Events
https://www.statnews.com/2026/05/31/squamous-cell-lung-cancer-akeso-summit-harmoni-asco-2026/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-9K_QeHmBTKiVP-VqCNkg3lSQlYPnOs74cV2rwrywwYrO9ZGNyS4Oxqoxf1APcYC0OGii-i_NBEGoDs4-icjir8jQyqAg&_hsmi=433538757&utm_content=433538757&utm_source=hs_email
Akeso and Summit's lung cancer drug shows worsening tumor-progression benefit
Chinese regulators on Friday approved an expanded use of ivonescimab, the PD-1/VEGF-targeting antibody made by the Chinese drugmaker Akeso, for the treatment of newly diagnosed squamous non-small cell lung cancer. The new prescription label, however, included a negative surprise: updated study results data showing a worsening tumor-progression benefit than previously reported.
The new disclosure is potentially bad news Summit Therapeutics, which is developing ivonescimab for the U.S. and European markets.
In an updated analysis of a study called HARMONi-6, the combination of ivonescimab and chemotherapy reduced the risk of tumor progression by 28%, down from 40% when the study was presented in May at the ASCO annual meeting. The improvement in overall survival shown by the ivonescimab-chemotherapy regimen has not changed, although it may with more mature data follow-up.
How this impacts Summit and its ongoing HARMONI-3 study of ivonescimab in the same squamous lung cancer patient population is uncertain. Last September, the company said patients in North America and Europe had seen their cancer return faster than those in China. As a result, an interim analysis of progression-free survival did not meet a prespecified endpoint. A final analysis is expected later this year.
How Low Does PSA Need to Go in Metastatic Prostate Cancer?
https://www.medscape.com/viewarticle/how-low-does-psa-need-go-metastatic-prostate-cancer-2026a1000rpa?ecd=WNL_trdalrt_pos1_ous_260817_etid8604317&uac=148436CN&impID=8604317
A real-world analysis suggests that achieving a prostate-specific antigen (PSA) level < 0.2 ng/mL within 9 months of starting androgen deprivation therapy (ADT)-based treatments was the PSA benchmark most consistently associated with improved overall survival in men with metastatic castration-sensitive prostate cancer. Achieving a PSA level < 0.2 ng/mL within 9 months of ADT initiation was associated with a significantly lower subsequent risk for death, while achieving 90% or greater PSA decline but not a PSA level < 0.2 ng/mL was not tied to improved overall survival.
domingo, 16 de agosto de 2026
Advances in X chromosome inactivation open therapeutic opportunities for X-linked genetic disorders
https://www.news-medical.net/news/20260803/Advances-in-X-chromosome-inactivation-open-therapeutic-opportunities-for-X-linked-genetic-disorders.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=rare_diseases_newsletter_10_august_2026
A new review explores how advances in understanding X chromosome inactivation (XCI) are creating potential therapeutic opportunities for a range of X-linked genetic disorders, including Rett syndrome, Fabry disease, Duchenne muscular dystrophy, hemophilia, and others. The article examines the molecular mechanisms that control X chromosome silencing, the impact of skewed XCI on disease severity, emerging methods to measure XCI patterns, and experimental approaches aimed at reactivating beneficial genes on the inactive X chromosome.
HMGCR emerges as a promising target for precision cancer therapies + + +
HMGCR emerges as a promising target for precision cancer therapies
https://www.news-medical.net/news/20260805/HMGCR-emerges-as-a-promising-target-for-precision-cancer-therapies.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
HMGCR emerges as a promising target for precision cancer therapiesA growing body of evidence is reshaping how scientists and clinicians understand the role of cancer metabolism, placing the enzyme HMGCR at the center of a rapidly evolving therapeutic landscape.
Tuft cell-like cancers exhibit unique biological characteristics across multiple organs
https://www.news-medical.net/news/20260806/Tuft-cell-like-cancers-across-multiple-organs-exhibit-unique-biological-characteristics.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
Tuft cell-like cancers exhibit unique biological characteristics across multiple organsA growing body of evidence suggests that a rare epithelial cell type known as the tuft cell may play a far more significant role in cancer than previously recognized.
Early radionuclide therapy delays progression in metastatic prostate cancer
https://www.news-medical.net/news/20260806/Early-radionuclide-therapy-delays-progression-in-metastatic-prostate-cancer.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
Earlier use of a targeted radionuclide therapy delays metastatic prostate cancer progression when administered as part of a trio of drugs, according to a phase 3 clinical trial led by Weill Cornell Medicine, NewYork-Presbyterian, Memorial Sloan Kettering Cancer Center and investigators at several other institutions worldwide.
Ferroptosis-based therapy shows promise for advanced prostate cancer treatment + + + + +
https://www.news-medical.net/news/20260806/Ferroptosis-based-therapy-shows-promise-for-advanced-prostate-cancer-treatment.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
A recent review explores the rapidly expanding field of ferroptosis-based therapy for prostate cancer, presenting ferroptosis as a promising strategy to overcome resistance in advanced and castration-resistant prostate cancer (CRPC). The authors synthesize current knowledge of ferroptosis mechanisms, biomarkers, therapeutic agents, combination strategies, and translational challenges, proposing a framework that links ferroptosis regulatory networks with precision medicine approaches.
Tadalafil may increase glaucoma risk in men with enlarged prostates
https://www.news-medical.net/news/20260804/Tadalafil-may-increase-glaucoma-risk-in-men-with-enlarged-prostates.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
An erectile dysfunction drug that is also used to treat symptoms like sudden urgency and frequency, which are associated with an enlarged prostate in men, may heighten the risk of the serious eye condition, glaucoma, finds a large comparative study published online in the British Journal of Ophthalmology.
Older men’s sexual priorities extend well beyond erectile function
https://www.news-medical.net/news/20260806/Older-mene28099s-sexual-priorities-extend-well-beyond-erectile-function.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
A web-based survey of 6,000 Japanese men aged 50 or older found that sexual arousal was the function they most wanted to preserve, while erection became more highly valued as erectile function worsened. Erection showed the earliest age-related decline pattern, whereas ejaculation was the function most often reported as lost in older age.
AI tool helps customize radiopharmaceutical therapy dosingAI tool helps customize radiopharmaceutical therapy dosing
https://www.news-medical.net/news/20260804/AI-tool-helps-customize-radiopharmaceutical-therapy-dosing.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
Radiopharmaceutical therapy (RPT) has made great strides over the last 50 years, having only recently received FDA approval to treat prostate cancer. But despite the progress-and billions of dollars of industry investments-one area that has lagged behind is dosing, which is still one-size-fits-all.
Study identifies a novel direct drug target for head and neck squamous cell carcinomaStudy identifies a novel direct drug target for head and neck squamous cell carcinoma
https://www.news-medical.net/news/20260806/Study-identifies-a-novel-direct-drug-target-for-head-and-neck-squamous-cell-carcinoma.aspx?utm_source=news_medical_newsletter&utm_medium=email&utm_campaign=prostate_cancer_newsletter_10_august_2026
Chemotherapy remains a cornerstone of treatment for head and neck cancers, the seventh-most common cancer type worldwide.
Blocking caspase-1 appears to reduce the risk of developing lung tumors
https://www.news-medical.net/news/20260814/Blocking-caspase-1-appears-to-reduce-the-risk-of-developing-lung-tumors.aspx
Every year, lung cancer kills more than 100,000 people in the United States. Smoking is the leading risk factor for lung cancer, but other environmental exposures can also contribute to the disease.
In an advance that could help prevent some of those lung cancer deaths, MIT researchers have shown that blocking an enzyme involved in lung inflammation appears to reduce the risk of developing tumors.
Saliva versus lesion swabs for PCR diagnosis of acute-phase clade Ib mpox in Uganda: a prospective matched hospital cohort study Jennifer Serwanga, PhDa,b,c Send email to jennifer.serwanga@mrcuganda.org ∙ Raymond Ernest Kaweesa, MBChBa ∙ Deborah Mukisa, BSca,b ∙ Geoffrey Odoch, BSca,b ∙ Brenda Nairuba, MBChBd ∙ Violet Ankunda, MScb ∙ et al.
Saliva versus lesion swabs for PCR diagnosis of acute-phase clade Ib mpox in Uganda: a prospective matched hospital cohort study
Jennifer Serwanga, PhDa,b,c Send email to jennifer.serwanga@mrcuganda.org ∙ Raymond Ernest Kaweesa, MBChBa ∙ Deborah Mukisa, BSca,b ∙ Geoffrey Odoch, BSca,b ∙ Brenda Nairuba, MBChBd ∙ Violet Ankunda, MScb ∙ et al.
https://www.thelancet.com/journals/lanmic/article/PIIS2666-5247(26)00050-9/fulltext?dgcid=raven_jbs_etoc_feature_lanmic
Widespread circulation of Alongshan virus in Austria and serological evidence for infection in humans: a nationwide molecular and serological observational study David M Florian, MDa ∙ Paul Jetzingera ∙ Anna-Margarita Schöttab ∙ Iris Medits-Weiss, PhDa ∙ Moritz Irgang, DVMc ∙ Cara L Ebert, PhDd,e ∙ et al.
Widespread circulation of Alongshan virus in Austria and serological evidence for infection in humans: a nationwide molecular and serological observational study
David M Florian, MDa ∙ Paul Jetzingera ∙ Anna-Margarita Schöttab ∙ Iris Medits-Weiss, PhDa ∙ Moritz Irgang, DVMc ∙ Cara L Ebert, PhDd,e ∙ et al.
https://www.thelancet.com/journals/lanmic/article/PIIS2666-5247(26)00059-5/fulltext?dgcid=raven_jbs_etoc_feature_lanmic
The human gut microbiome: still more questions than answers The Lancet Microbe +...
The human gut microbiome: still more questions than answers
The Lancet Microbe
https://www.thelancet.com/journals/lanmic/issue/vol7no8/PIIS2666-5247(26)X2007-9
Human versus analogue insulin for children and young adults with type 1 diabetes in low-resource settings (HumAn-1): a multicentre, open-label, randomised controlled trial
Human versus analogue insulin for children and young adults with type 1 diabetes in low-resource settings (HumAn-1): a multicentre, open-label, randomised controlled trial
Dr Jing Luo, MDa Send email to luoj@pitt.edu ∙ Sylvia Kehlenbrink, MDb ∙ Chung-Chou H Chang, PhDa,c ∙ Christina M Lalama, MSa ∙ Jill Kirsch, MSa ∙ Éimhín Ansbro, MDd ∙ et al.
https://www.thelancet.com/journals/landia/article/PIIS2213-8587(26)00097-5/abstract?dgcid=raven_jbs_etoc_feature_landia
Whole-population trends in obesity across dimensions of inequality in England, 2019–25: a retrospective, longitudinal cohort study of 54 million adults
Whole-population trends in obesity across dimensions of inequality in England, 2019–25: a retrospective, longitudinal cohort study of 54 million adults
Robert A Fletcher, MSca,b,d,f Send email to raf69@medschl.cam.ac.uk ∙ Nathalie Conrad, DPhilg,h ∙ Patrick Rockenschaub, PhDi ∙ Stelios Boulitsakis Logothetis, MEnga,b ∙ Brendon L Neuen, PhDf,j ∙ Fionna Chalmers, MSce ∙ et al.
https://www.thelancet.com/journals/landia/article/PIIS2213-8587(26)00120-8/fulltext?dgcid=raven_jbs_etoc_feature_landia
Soaring GLP-1 receptor agonist use The Lancet Diabetes & Endocrinology +...
Soaring GLP-1 receptor agonist use
The Lancet Diabetes & Endocrinology
https://www.thelancet.com/journals/landia/issue/vol14no9/PIIS2213-8587(26)X2007-1
Blended mobile health and wellness coaching enhances student engagement in mental health care Michael Reifman [1] , Xiao Cao [1] , Michelle L. Rozwadowski [1] , Jaeryeong Kim [1] , Maxwell Kluge [1] , Uma L. Subrayan [1] , Marsha Benz [2] , Amanda Reis [2] , Jevon Moore [2] , Kerby Shedden [3] , Sung Won Choi* [1]
https://www.academia.edu/2997-9196/3/2/10.20935/MHealthWellB8298
Introduction: Mental health challenges are common among college students, highlighting the need for scalable approaches that aim to reduce distress and support well-being. mHealth tools may complement campus services, though sustained engagement may be difficult. This study sought to discover how a blended intervention combining an mHealth positive psychology intervention (PPI) app with optional wellness coaching might impact student well-being.
Materials and methods: In this single-arm pilot study, 28 students at a public university were given access to a PPI app (Roadmap 2.0) with mood tracking, a Fitbit® wearable device, and optional wellness coaching. Data sources included PROMIS® surveys at baseline and monthly follow-ups, daily mood ratings, app engagement logs, wearable-derived activity metrics, coaching attendance, and optional exit interviews. Analyses were descriptive and exploratory.
Results: From baseline to exit, participants showed descriptive increases in PROMIS® global mental health and positive affect and decreases in depression, anxiety, fatigue, and anger. App engagement declined over time. In exploratory models, app engagement was lower among participants reporting greater psychosocial resources or support. Mood ratings were higher in the days following PPI activity completion, and PPI activity users were observed to have higher mood ratings over time. Interviews supported perceived app–coaching synergy and identified barriers to sustained engagement.
Conclusions: A blended PPI mHealth app plus wellness coaching appears feasible in a real-world college setting and was associated with favorable descriptive trends in mental health and well-being outcomes. Controlled studies are needed to evaluate efficacy and assess app versus coaching contributions.
https://www.academia.edu/journals/academia-mental-health-and-well-being/articles?source=journal-top-nav
'Tony' Review: Anthony Bourdain's coming-of-age story unfolds in charming dramedy (+...)
'Tony' Review: Anthony Bourdain's coming-of-age story unfolds in charming dramedy
Fox News Digital reviews A24's Anthony Bourdain coming-of-age dramedy "Tony" starring Dominic Sessa, Emilia Jones, Leo Woodall and Antonio Banderas. Directed by Matt Johnson.
https://www.foxnews.com/lifestyle?lid=4u0xwkbm36dv
sábado, 15 de agosto de 2026
A Body at War: New Immune Reset By Ivanhoe Broadcast News on August 13, 2026Family
DENVER, Colo. (Ivanhoe Newswire) — More than 50 million Americans live with autoimmune diseases — conditions in which the immune system mistakenly attacks the body it’s supposed to protect. For many, treatment can mean years of medications to quiet that attack. But now, doctors are testing something far more aggressive: ways to reset or even replace the immune system itself.
https://www.ivanhoe.com/family-health/a-body-at-war-new-immune-reset/
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