sábado, 21 de marzo de 2026

Family pleads for help as teen faces life-threatening bone marrow failure By Melissa Rudy Fox News Published March 20, 2026 3:22pm EDT

Family pleads for help as teen faces life-threatening bone marrow failure Mixed ancestry makes finding a lifesaving stem cell match far more difficult Melissa Rudy By Melissa Rudy Fox News Published March 20, 2026 3:22pm EDT https://www.foxnews.com/health/family-pleads-help-teen-faces-life-threatening-bone-marrow-failure

Wellness expert reveals surprising health benefits of daily cold exposure: 'Huge difference' +++

Wellness expert reveals surprising health benefits of daily cold exposure: 'Huge difference' And more of this week's top health stories, right to your inbox By Fox News Staff Fox News Published March 20, 2026 4:32pm EDT | Updated March 20, 2026 4:42pm EDT https://www.foxnews.com/health/wellness-expert-reveals-surprising-health-benefits-daily-cold-exposure-huge-difference Wim Hof breaks down his three-pillar method for more energy, less stress "The Iceman" Wim Hof explains how exposure to low temperatures, breathing and focused mindset work together to support physical performance and improve stress response. https://www.foxnews.com/video/6391178641112 'Ice man' shares one daily habit that could boost wellness and brainpower Study shows benefits of Wim Hof Method, which combines cold exposure and breathwork Angelica Stabile By Angelica Stabile Fox News Published March 19, 2026 6:00am EDT https://www.foxnews.com/health/ice-man-shares-one-daily-habit-could-boost-wellness-brainpower

Stopping Ozempic? New study reveals surprising weight regain results after GLP-1s By Khloe Quill Fox News Published March 18, 2026 1:52pm EDT

Stopping Ozempic? New study reveals surprising weight regain results after GLP-1s Study of nearly 8,000 patients found average weight regain of just 0.5% one year after stopping medications By Khloe Quill Fox News Published March 18, 2026 1:52pm EDT https://www.foxnews.com/health/stopping-ozempic-new-study-reveals-surprising-weight-regain-results-after-glp-1s

Needle-free diabetes management could be on the horizon, study suggests By Angelica Stabile Fox News Published March 20, 2026 6:00am EDT

Needle-free diabetes management could be on the horizon, study suggests Experimental insulin pill delivers significant blood sugar reductions in early testing Angelica Stabile By Angelica Stabile Fox News Published March 20, 2026 6:00am EDT https://www.foxnews.com/health/needle-free-diabetes-management-could-horizon-study-suggests

Widespread habit may raise colorectal cancer risk more than you think By Melissa Rudy Fox News Published March 21, 2026 9:00am EDT

Widespread habit may raise colorectal cancer risk more than you think Even moderate alcohol consumption tied to higher colorectal cancer risk over time Melissa Rudy By Melissa Rudy Fox News Published March 21, 2026 9:00am EDT https://www.foxnews.com/health/widespread-habit-may-raise-colorectal-cancer-risk-more-than-you-think

Deadly meningitis outbreak prompts college students to call for campus shutdown By Melissa Rudy Fox News Published March 19, 2026 2:59pm EDT

Deadly meningitis outbreak prompts college students to call for campus shutdown Petition calls for closure following 15 confirmed cases and 2 deaths in UK county Melissa Rudy By Melissa Rudy Fox News Published March 19, 2026 2:59pm EDT https://www.foxnews.com/health/deadly-meningitis-outbreak-prompts-college-students-call-campus-shutdown

The Right Workout For Your Personality By Ivanhoe Broadcast News on March 27, 2026

Orlando, Fla. (Ivanhoe Newswire) — March is a time when many people start to rethink their health, especially as we focus on heart and overall wellness. Experts say staying active is one of the most powerful ways to protect your health. But it turns out how you exercise may depend on who you are. https://www.ivanhoe.com/?p=36850&preview=1&_ppp=7459e723cf

Gramping: Spring Break For Teens & Grandparents By Ivanhoe Broadcast News on March 26, 2026

Orlando, Fla. (Ivanhoe Newswire) — When you think of family Spring break vacations with your teen, you might think of beach trips or theme park getaways. Now, many families are opting for “gramping,” it’s when grandparents and grandchildren vacation together and leave the parents at home. https://www.ivanhoe.com/?p=36847&preview=1&_ppp=785e1fd9e2

Scratch Season: Hidden Health Risks For Pets This Spring By Ivanhoe Broadcast News on March 25, 2026

Orlando, Fla. (Ivanhoe Newswire) — Spring fever isn’t just hitting people, it’s hitting pets. New national forecasts show flea and tick season is starting earlier and lasting longer across much of the U.S. Veterinarians are also seeing a spike in dog allergies with symptoms many pet parents don’t realize are allergy related. https://www.ivanhoe.com/?p=36844&preview=1&_ppp=5c19044ae9

Privacy Check: Who’s Tracking You Now? By Ivanhoe Broadcast News on March 24, 2026

Orlando, Fla. (Ivanhoe Newswire) — Smart devices are everywhere, in our pockets, in our living rooms, even in our cars! But as more of our lives go digital, experts warn the data these devices collect may be far more extensive and far more personal than many people realize. Privacy surveys show more than half of consumers don’t trust smart devices to protect their personal data, and nearly three-quarters worry their information is used without their permission. https://www.ivanhoe.com/?p=36841&preview=1&_ppp=b4c50c65c3

Clean Smarter This Spring: Protecting Your Family’s Health At Home By Ivanhoe Broadcast News on March 23, 2026

Orlando, Fla. (Ivanhoe Newswire) — Surveys show about 80 percent of Americans Spring clean every year. But while many families focus on closets and garages, experts say spring is also the perfect time to clean out hidden exposures that can affect your child’s health. From everyday plastics to cleaning products and indoor air. https://www.ivanhoe.com/?p=36838&preview=1&_ppp=dc0e287182

LaMer growth of iron nanoparticles for magnetic particle imaging Aleia G. Williams [1] , Willem Graham [1] , Sydney Henriques [2] , Todd D. Giorgio [2] , Charles E. Johnson [3] , Jacqueline A. Johnson* [1,3]

https://www.academia.edu/2997-2027/2/4/10.20935/AcadMatSci8015 Abstract Magnetic particle imaging (MPI) is a relatively new imaging technique that uses magnetic nanoparticles as tracers to generate a signal. Compared to magnetic resonance imaging, MPI detects the magnetization of nanoparticles directly rather than relying on secondary effects of magnetic resonance relaxation times from protons. Iron oxide nanoparticles with diameters in the 25–30 nm range are among the most commonly used MPI tracers. However, we hypothesize that further improvement in tracers can be achieved by utilizing pure iron nanoparticles. From physics-based models, iron nanoparticles with diameters of at least 20 nm are needed to enhance MPI performance. Previous studies have used thermal decomposition of organic iron precursors to create iron nanoparticles between 5 and 15 nm in diameter, but few studies have successfully created iron nanoparticles of larger sizes. Therefore, we investigated the use of an extended LaMer mechanism to create larger iron nanoparticle sizes for potential MPI applications. Continuous addition of Fe(CO)5 as the precursor using a thermal decomposition method was used. Three injection speeds of the iron precursor were tested, which included 100, 50, and 25 μl/min to test if injection rates affected the overall particle growth and size. In addition, three different surfactants, an oleylamine/oleic acid (OAm/OA) mixture, hexadecylamine (HDA), and octadecylamine (ODA), were used in order to test if the surfactants affected the size, morphology, and composition of the nanoparticles. Larger nanoparticle diameters up to 24 and 26 nm were observed at lower injection speeds when HDA and ODA were used, respectively. However, samples using OAm/OA mixtures remained around 15.5 nm at all injection speeds. In addition, OAm/OA samples displayed high magnetic saturation values up to 138 emu/g, but samples using HDA and ODA displayed lower magnetic saturation values up to 46 emu/g and 38 emu/g, respectively. https://www.academia.edu/journals/academia-materials-science/articles?source=journal-top-nav

RSV in the Spotlight: Why Is There an Urgency to Protect High-Risk Populations Through Immunization? Authors: Oliver Cornely, MD; Jane Marie Barratt, PhD, MSc, BSc

https://www.medscape.org/viewarticle/rsv-spotlight-why-there-urgency-protect-high-risk-2026a10002lr?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260320-OUS-HONC-rsv-spotlight-why-there-urgency-protect-high-risk-2026a10002lr-cta&sso=true

Most Read Papers - Cancer Science & Oncogenesis +...

https://read.qxmd.com/collection/1133?ecd=wnl_readmost_260320 Adhesion forces between macrophages and cancer cells promote early tumor development. Biochemical mechanisms of macrophage-driven tumor promotion are well documented, but the contribution of physical forces to early tumor development remains poor.. Mar 17, 2026: Proceedings of the National Academy of Sciences of the United States of America

Current and Future Oral SERD Strategies in the Management of ER-Positive/HER2-Negative MBC: Analyzing the Clinical Data Authors: Komal Jhaveri, MD, FACP, FASCO; Shaheenah Dawood, MD, MPH

https://www.medscape.org/viewarticle/current-and-future-oral-serd-strategies-management-er-2026a100022b?page=1&uac=148436CN&sso=true&src=mkmcmr_driv_stan_mscpedu_260320-OUS-HONC-current-and-future-oral-serd-strategies-management-er-2026a100022b-cta

FDA approves nivolumab with chemotherapy for previously untreated Hodgkin lymphoma

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-chemotherapy-previously-untreated-hodgkin-lymphoma?utm_medium=email&utm_source=govdelivery

Challenges and Opportunities for CAR T-Cell Therapies to Treat Autoimmune Rheumatic Diseases Authors: Eric Morand, MD, PhD; David Simon, MHBA; Fabian Müller, MD; Gerhard Krönke, MD, MHBA

https://www.medscape.org/viewarticle/1003043?src=mkmcmr_driv_stan_mscpedu_260320-OUS-HONC-1003043-cta&uac=148436CN&sso=true

Dose-Dense Chemotherapy Enables Elimination of RT for the Majority of Low-Risk Pediatric Hodgkin Lymphomas: PHC Study HOD08 Authors: Jamie E. Flerlage, MD, MS; Angela M. Feraco, MD, MMSc; Yiwang Zhou, PhD; Ying Zheng, MS; Jia Liang, PhD; John T. Lucas, Jr, MD, MS; Alison M. Friedmann, MD, MSc; Howard J. Weinstein, MD; Torunn I. Yock, MD; Barry Shulkin, MD, MBA; Sue C. Kaste, DO; Lianna J. Marks, MD; Matthew J. Ehrhardt, MD, MS; Stephanie B. Dixon, MD, MPH; Scott Howard, MD; Pedro de Alarcon, MD; Sandra Luna-Fineman, MD; Amy E. Geddis, MD, PhD; Eric C. Larsen, MD; Karen Marcus, MD; Amy L. Billett, MD; Sarah S. Donaldson, MD; Melissa M. Hudson, MD; Monika L. Metzger, MD, MS; Matthew J. Krasin, MD; Michael P. Link, MD

https://www.medscape.org/viewarticle/1003274?impID=8203361&uac=148436CN&sso=true&src=wnl_tpal_260320_mscpedu

Clinical Perspectives on the Use of PI3K/AKT Pathway Inhibitors in the Management of HR+/HER2− Metastatic Breast Cancer Authors: Peter Schmid, MD, PhD, FRCP; Kevin Kalinsky, MD, MS, FASCO; Frédérique Penault-Llorca, MD, PhD

https://www.medscape.org/viewarticle/clinical-perspectives-use-pi3k-akt-pathway-inhibitors-2026a100087h?page=1&uac=148436CN&sso=true&src=wnl_tpal_260320_mscpedu&impID=8203361

Therapeutic Evolution in mHSPC: Analyzing Emerging Combinations and Outcomes Authors: Rana R. McKay, MD, FASCO; Alicia K. Morgans, MD, MPH, FASCO; David Morris, MD, FACS

https://www.medscape.org/viewarticle/therapeutic-evolution-mhspc-analyzing-emerging-combinations-2026a100087o?page=1&sso=true&uac=148436CN&impID=8203361&src=wnl_tpal_260320_mscpedu

Stress can cause eczema to flare up – now we know why Scientists have identified the neurons that worsen the condition during stress. By Miryam Naddaf

https://www.nature.com/articles/d41586-026-00876-3?utm_source=Live+Audience&utm_campaign=c9d0ea9a74-nature-briefing-daily-20260320&utm_medium=email&utm_term=0_-33f35e09ea-50432164 For people with eczema, stress can trigger flare-ups and worsen their itchy rashes. But the link between stress and skin inflammation has been unclear.

viernes, 20 de marzo de 2026

The importance of ever-evolving phase 1 trials in oncology Andreas Domen* [1,2] , Pieterjan Vanclooster [1,2] , Laure-Anne Teuwen [1,2] , Bernd Dekeyser [1] , Timon Vandamme [1,2] , Hans Prenen [1,2]

https://www.academia.edu/2998-7741/3/1/10.20935/AcadOnco8203 Abstract Phase 1 clinical trials are a critical step in oncology drug development. Historically, these first-in-human trials focused on dose–toxicity profiling in heterogeneous populations. With the emergence of novel anticancer therapies, trial design has shifted toward model-based and adaptive dose-escalation designs, improving dose optimization, and now incorporates biomarker-driven patient selection and multiple objectives, including early efficacy assessment, which accelerates drug development. Hence, phase 1 clinical trials have undergone substantial transformation over recent decades, with a marked impact on response rates and phase 1 units that conduct these trials. By transforming early-stage drug discovery and target identification as well as predicting drug efficacy and toxicity, the implementation of artificial intelligence into early-phase cancer research will further accelerate the discovery of novel anticancer drugs and significantly transform today’s phase 1 trials, ultimately resulting in an increase in phase 1 clinical trials in the near future. In this narrative review, we address the importance of clinical phase 1 trials for oncology drug development, highlight their continuous evolution over the past decades to date, and discuss how this affects phase 1 units and their investigators, as well as exploring the impact of artificial intelligence on phase 1 trials in the coming years. https://www.academia.edu/journals/academia-oncology/articles?source=journal-top-nav

Advancing Personalized Care for Prostate Cancer: Patient-Centered Solutions for mHSPC and nmCRPC Authors: Alicia K. Morgans, MD, MPH, FASCO; Christian Gratzke, MD; Fred Saad, MD, FRCS, FCAHS; Tony Collier, BEM

https://www.medscape.org/viewarticle/advancing-personalized-care-prostate-cancer-patient-centered-2025a1000uyb?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260319-OUS-HONC-advancing-personalized-care-prostate-cancer-patient-centered-2025a1000uyb-cta&sso=true

Beyond the Update in Advanced/Recurrent Endometrial Cancer: How Evidence Is Shaping New Guidelines Authors: Nicole Concin, MD, PhD; Els Van Nieuwenhuysen, MD, PhD; Xavier Matias-Guiu, MD, PhD

https://www.medscape.org/viewarticle/beyond-update-advanced-recurrent-endometrial-cancer-how-2026a10006up?page=1?src=mkmcmr_reeng_recap_mscpedu_activity&sso=true&uac=148436CN thank you for your recent participation in the activity: Beyond the Update in Advanced/Recurrent Endometrial Cancer: How Evidence Is Shaping New Guidelines Below are some key learning points to help reinforce the impact of this activity. ☑ ESGO–ESTRO–ESP 2025 Guidelines (staging and risk classification) • Guidelines integrated FIGO 2023 staging and molecular classification (POLE, MMR status, P53) to refine risk groups and drive treatment selection ☑ Clinical trials • Four phase 3 trials (RUBY, NRG‑GY018, DUO‑E, AtTEnd) showed adding immune checkpoint inhibitors (ICI) to platinum‑based chemo greatly improves outcomes in dMMR advanced/recurrent endometrial cancer (HRs 0.28–0.42); however, the addition of PARP inhibitors did not offer an increased benefit (RUBY, DUO-E) • ​In non‑dMMR disease, ICI with chemo only provided moderate PFS benefit; adding PARP in maintenance (RUBY Part 2, DUO‑E) further improved PFS (HRs 0.57–0.63) with manageable additional toxicity ☑ ​ESGO–ESTRO–ESP 2025 Guidelines (therapy) • ​Key updated recommendation includes: ◦ ​First‑line dMMR = chemo + ICI, followed by ICI maintenance ◦ ​Non‑dMMR symptomatic disease chemo + ICI ± PARP maintenance ☑ Complete molecular testing should be performed at diagnosis (POLE, MMR IHC, P53 IHC/NGS if ambiguous) and retesting at progression when possible as the results guide treatment decisions

Ending the diagnostic odyssey: How Europe can lead the way in rare disease diagnosis EURORDIS-Rare Diseases Europe

https://www.linkedin.com/pulse/ending-diagnostic-odyssey-how-europe-can-lead-way-rare-disease-quv2e/ For many people living with rare diseases, the hardest part of their illness isn’t the treatment. It’s the wait for a diagnosis. Symptoms appear. Appointments follow. Tests are run. Specialists are consulted. And still, for what can be years, there are no answers.

Award-winning project on Tuberous Sclerosis Complex

https://drive.google.com/file/d/19ZE0Ol3qDhCtCOY1ImYgJUKWDalg3zNS/view?pli=1 Award-winning project on Tuberous Sclerosis Complex Catarina Loução and a group of students created a scientific magazine dedicated to Tuberous Sclerosis Complex (TSC) as part of the competition “Genetics & Rare Diseases – One Vision, Multiple Perspectives”, promoted by the Department of Human Genetics at the National Institute of Health Doutor Ricardo Jorge in Portugal. The project was awarded first prize and aims to improve science communication and increase awareness of TSC among the general public, students, and the wider rare disease community. The team also presented their work at the 6th World Rare Disease Day Conference on 28 February in Portalegre, Portugal. The magazine is available in Portuguese and English, offering accessible but detailed insights into this rare condition. Read the magazine!

Pre-training – Scientific Innovation and Translational Research

https://openacademy.eurordis.org/courses/pre-training-scientific-innovation-and-translational-research/ Scientific Innovation and Translational Research Eager to learn more about the rare disease research and diagnosis pathway? The EURORDIS Open Academy offers a course on how medicines are brought to market, forming part of the required pre-training for the Open Academy School on Scientific Innovation and Translational Research. Enrol now! https://openacademy.eurordis.org/

Rare Diseases, from A to Z. Volume 2 by Matthieu MERON (in collaboration with Plateforme Expertise Maladies Rares - Bourgogne-Franche-Comté, Prof. Laurence FAIVRE, Sonia GOERGER, Amandine BAURAND)

https://www.fondation-ipsen.org/book-lab/rare-diseases-from-a-to-z-volume-2/ For Rare Disease Day, Fondation Ipsen published the second volume of their book, Rare Diseases, from A to Z. Created with the Rare Disease Expertise Platform of Bourgogne-Franche-Comté, the book presents a range of rare conditions through clear descriptions paired with symbolic illustrations by artist Matthieu Méron. Combining scientific detail with artistic storytelling, it aims to improve public understanding, support education, encourage earlier diagnosis, and help increase recognition and visibility for people living with rare diseases and their families. http://www.pemr-bfc.fr/

Incorporating cost considerations into shared decision-making: A cross-country comparison among Hungary, France, and Bulgaria

Incorporating cost considerations into shared decision-making: A cross-country comparison among Hungary, France, and Bulgaria Guenka Petrova, Maria Kamusheva, Konstantin Tachkov, Maria Dimitrova, Teodora Chamova, Yoana Seitaridu, Vera Stoeva, Martin Donchev, Bertalan Nemeth, Zsuzsanna Ida Petykó, Anna Bögös, Dalma Hosszu, Julien Delaye, François Houÿez Abstract This study aims to compare the patients’ and healthcare professionals’ (HCPs) opinions about the necessity and acceptance of discussing the cost of treatment as part of shared decision-making (SDM) in three healthcare jurisdictions – Bulgaria, Hungary, and France. https://pharmacia.pensoft.net/article/185701/ This article, co-authored by François Houÿez, our Director of Information & Access to Therapies, explores whether treatment costs should be discussed between patients and clinicians as part of shared decision-making. Conducted within the now-completed HTx (Next Generation Health Technology Assessment) project, the study presents findings from focus groups involving patients and healthcare professionals in Hungary, France, and Bulgaria. https://www.eurordis.org/projects/htx-next-generation-hta/

13th European Conference on Rare Diseases and Orphan Products (ECRD 2026) 3 & 4 June 2026 in Prague  and online

https://www.rare-diseases.eu/

Increase your knowledge and skills in the rare disease space

https://openacademy.eurordis.org/ The EURORDIS Open Academy accompanies patient advocates by offering rare disease-specific comprehensive training programmes that empower advocates with the knowledge, skills and confidence they need to engage with different stakeholders as equal partners. Our Schools, supported by ERDERA, offer a blended learning approach that includes e-learning courses, webinars, in-person training, as well as research visits and networking opportunities.

Rare Diseases Forum 2026

https://events.theparliamentmagazine.eu/event/rare-diseases-forum/ The Rare Diseases Forum 2026 comes at a crucial time for EU health policy. With over 30 million Europeans affected, momentum is growing for a more coordinated response. In 2025, the European Parliament called for a comprehensive EU Action Plan on Rare Diseases, urging stronger Member State commitment. Upcoming reforms to EU pharmaceutical law and the HTA regulation will reshape access to orphan drugs.

Podcast: Rare on Air

https://www.eurordis.org/rare-on-air/ Rare Disease Day bonus series Listen to the final episodes from our bonus podcast series Rare on Air Stories, which highlight powerful lived experiences from people impacted by rare diseases worldwide. In the latest episode, Stephanie shares her journey as a parent navigating her daughter's rare condition, with determination and strength. Ren reflects on living with an extremely rare disease, and how sharing her experience has helped her build connection and support

Rare Disease Day 2026 highlights global inequities and amplifies youth voices in call for lasting change

https://www.rarediseaseday.org/news/rare-disease-day-2026/ Rare Disease Day On 28 February, Rare Disease Day united communities worldwide to raise awareness of the realities faced by over 300 million people living with a rare disease. This year’s campaign, “More Than You Can Imagine,” highlighted the scale of global inequalities and the need for greater equity in diagnosis, care, research, and support. Read more!

Union list of critical medicines The Union list of critical medicines helps prevent issues with the supply and availability of medicines at European Union (EU) level.

https://www.ema.europa.eu/en/human-regulatory-overview/post-authorisation/medicine-shortages-availability-issues/availability-medicines-during-crises/union-list-critical-medicines Critical medicines On 4 March, we held a webinar on how patient organisations can effectively comment on the Union list of Critical Medicines, to make their voices heard and strengthen access to essential treatments. Led by our Director of Treatment Information and Access, François Houyez, the webinar presented advice and practical experiences on the process of consultations and ensuring continued availability of critical medicines. Webinar: How can patient organisations comment on the Union List of Critical Medicines February 2026 https://www.eurordis.org/webinar-union-list-of-critical-medicines/

EURORDIS supports EU Member States’ efforts to secure reintroduction of health NGOs’ operating grants March 2026

EURORDIS supports EU Member States’ efforts to secure reintroduction of health NGOs’ operating grants March 2026 4 March 2026, Brussels – EURORDIS–Rare Diseases Europe, together with fellow members of the EU4Health Civil Society Alliance, reiterates its call for the European Commission to reinstate operating grants for health non-governmental organisations, as political momentum grows among EU Member States for their return from 2026 onwards. https://www.eurordis.org/eurordis-supports-member-states-efforts-operating-grants-reintroduction/ Also this month, France sent a non-paper to the European Commission, formally endorsed by 12 other Member States, calling for the reintroduction of operating grants under EU4Health from 2026. EURORDIS welcomed France's initiative, with Virginie Hivert, our Acting Chief Executive Officer, reiterating that "stable partnerships strengthen the quality of EU health policy."

EURORDIS welcomes European Parliament draft report calling for a European Action Plan on Rare Diseases March 2026

https://www.europarl.europa.eu/doceo/document/SANT-PR-785192_EN.pdf EURORDIS welcomes European Parliament draft report calling for a European Action Plan on Rare Diseases March 2026 5 March 2026, Brussels – EURORDIS–Rare Diseases Europe welcomes the draft report presented by MEP Nicolás González Casares, Member of the European Parliament Committee on Public Health (SANT), calling on the European Commission to propose legislation establishing a European Rare Disease Action Framework. https://www.eurordis.org/response-draft-proposal-for-action-plan/ Earlier this month, Nicolás González Casares, Member of the European Parliament Public Health (SANT) Committee, presented to the Committee a draft report urging the European Commission to legislate for a European Action Plan. "We are very happy with this draft report," responded Valentina Bottarelli, our Head of Policy & Public Affairs. "The scale of the challenge demands a coordinated European approach."

Ending the diagnostic odyssey: How Europe can lead the way in rare disease diagnosis March 2026

Ending the diagnostic odyssey: How Europe can lead the way in rare disease diagnosis March 2026 For many people living with rare diseases, the hardest part of their illness isn’t the treatment. It’s the wait for a diagnosis. Symptoms appear. Appointments follow. Tests are run. Specialists are consulted. And still, for what can be years, there are no answers. https://www.eurordis.org/how-europe-can-lead-way-rare-disease-diagnosis/ Despite numerous appointments, tests, and specialist consultations, many people living with a rare disease wait years without answers – a journey often referred to as the “diagnostic odyssey”. https://www.rare-diseases.eu/ This challenge will be among those explored at our European Conference on Rare Diseases and Orphan Products (ECRD 2026), taking place on 3–4 June in both Prague and online, particularly as part of the conference's Diagnosis, Research and Prevention track. Our latest article explores the scale of the diagnostic odyssey and outlines how Europe could set the global benchmark for improving the diagnostic journey.

Redefining Standards in Bladder Cancer: The Expanding Role of Immunotherapy Authors: Andrea Necchi, MD; James W. F. Catto, MB ChB, PhD, FRCS; Maria De Santis, MD

https://www.medscape.org/viewarticle/redefining-standards-bladder-cancer-expanding-role-2025a1000ws0?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260319-OUS-HONC-redefining-standards-bladder-cancer-expanding-role-2025a1000ws0-cta&sso=true

American Academy of Allergy, Asthma & Immunology (AAAAI) 2026 Annual Meeting

https://www.medscape.com/c25/p14//american-academy-allergy-asthma-immunology-aaaai-2026-annual-2026a10003e3?ecd=wnl_conf_allergy_AAAAI-non-spon_260320_mscpedit_etid8197425&uac=148436CN&impID=8197425

A Lump in the Throat: Thyroid Cancer Ali Ahmad, MD March 12, 2026 ++++

A Lump in the Throat: Thyroid Cancer Ali Ahmad, MD https://reference.medscape.com/p11/lump-throat-thyroid-cancer-2026a10006w7?ecd=wnl_critimg_260320_mscpref_etid8194596&uac=148436CN&impID=8194596 March 12, 2026 Thyroid cancer is the most common malignancy of the endocrine system and the 12th most common cancer in the United States. The incidence of thyroid cancer has been increasing over the last several decades. Thyroid cancer occurs nearly three times more often in women than in men. ATA Issues New Guidelines for Differentiated Thyroid Cancer Kerry Dooley Young https://www.medscape.com/viewarticle/ata-issues-new-guidelines-differentiated-thyroid-cancer-2025a1000o8x?ecd=wnl_critimg_260320_mscpref_etid8194596&uac=148436CN&impID=8194596 September 12, 2025 New guidelines from the American Thyroid Association (ATA) for managing adults with differentiated thyroid cancer (DTC) include several recommendations aimed at de-escalating treatment where possible, while also suggesting further work is needed to address financial toxicity of this disease. Thyroid Nodules and BCC: Don’t Overtreat Older Adult Patients Douglas S. Paauw https://www.medscape.com/viewarticle/thyroid-nodules-and-bcc-dont-overtreat-older-adult-patients-2025a1000t33?ecd=wnl_critimg_260320_mscpref_etid8194596&uac=148436CN&impID=8194596 October 27, 2025 Adding to the Toolbox: Thyroid Artery Embolization Kaniksha Desai, MD; Juan C. Camacho, MD https://www.medscape.com/viewarticle/1003208?ecd=wnl_critimg_260320_mscpref_etid8194596&uac=148436CN&impID=8194596 February 24, 2026

jueves, 19 de marzo de 2026

Alcohol health risks depend on beverage type and consumption habits

https://www.news-medical.net/news/20260319/Alcohol-health-risks-depend-on-beverage-type-and-consumption-habits.aspx While high alcohol intake has been associated with worse health outcomes regardless of the type of alcohol consumed, the potential impacts of low to moderate alcohol intake appear to vary by beverage type, according to a study being presented at the American College of Cardiology's Annual Scientific Session (ACC.26).

Eleuthero: Siberian Ginseng Benefits for Stress, Energy, and Fatigue

https://www.news-medical.net/health/Eleuthero-Siberian-Ginseng-Benefits-for-Stress-Energy-and-Fatigue.aspx From its traditional use for fatigue and weakness to modern research on inflammatory pathways, metabolism, and neuroprotection, eleuthero is emerging as a biologically intriguing adaptogen whose promise remains to be substantiated by stronger clinical evidence.

Schisandra Berry Benefits, Side Effects, Dosage, and Uses

https://www.news-medical.net/health/Schisandra-Berry-Benefits-Side-Effects-Dosage-and-Uses.aspx Schisandra berry is a traditional medicinal fruit rich in lignans and other bioactive compounds that show antioxidant, anti-inflammatory, and hepatoprotective potential. Current evidence is promising, but most benefits remain supported mainly by preclinical studies rather than large human trials.

How GLP-1 Weight-Loss Drugs Affect Appetite, Mood, and Behavior

https://www.news-medical.net/health/How-GLP-1-Weight-Loss-Drugs-Affect-Appetite-Mood-and-Behavior.aspx The widely used obesity and diabetes GLP-1 drugs may do far more than reduce weight. Emerging evidence suggests they alter food cue reactivity, reward signaling, and addictive behaviors, while leaving key questions about mood and emotional eating unresolved.

Study finds a clear link between ultraprocessed food and heart disease risk

https://www.news-medical.net/news/20260318/Study-finds-a-clear-link-between-ultraprocessed-food-and-heart-disease-risk.aspx In more than 6,500 US adults free of cardiovascular disease at baseline, researchers found that higher ultraprocessed food intake tracked with a higher long-term risk of ASCVD, with a stronger association in Black participants.

Survivorship: A Sustainable Resource

https://www.medscape.com/mtv/cancer-survivorship-s03/e06?src=hpcarousel

Hidradenitis Suppurativa Associated With Elevated Risks for Multiple Cancer Types Edited by Vineeta Teotia Medscape UK March 19, 2026

https://www.medscape.com/viewarticle/hidradenitis-suppurativa-associated-elevated-risks-multiple-2026a100084v

Female Survival Advantage Narrows in Midlife Due to Cancer Will Pass March 19, 2026

https://www.medscape.com/viewarticle/female-survival-advantage-narrows-midlife-due-cancer-2026a10008fm

GLP-1 Therapy: Cancer Benefit or Bias? Maurie Markman, MD March 19, 2026

https://www.medscape.com/viewarticle/glp-1-therapy-cancer-benefit-or-bias-2026a10005zd

A Call to Action: Addressing the Burden of RSV in Adults Through Prevention With Vaccines Authors: Peter Openshaw, CBE, PhD, FRCP, FMedSci; Ann R. Falsey, MD

https://www.medscape.org/viewarticle/call-action-addressing-burden-rsv-adults-through-prevention-2025a1000x9g?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260319-OUS-HONC-call-action-addressing-burden-rsv-adults-through-prevention-2025a1000x9g-cta&sso=true

Charting New Frontiers in Advanced Breast Cancer Milestones, Missions, and Mastery EBAC ACCREDITED Wednesday, 15 April 2026 | 17:30 - 19:00 CET

https://na.eventscloud.com/website/93510/

Precision Oncology Meets RAS: Innovations in Targeting RAS-Addicted Solid Tumors Authors: Kathryn C. Arbour, MD; Aparna R. Parikh, MD, MS

https://www.medscape.org/viewarticle/precision-oncology-meets-ras-innovations-targeting-ras-2025a1000x8k?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260318-OUS-HONC-precision-oncology-meets-ras-innovations-targeting-ras-2025a1000x8k-cta&sso=true

Optimizing Outcomes With Radiopharmaceuticals in mCRPC Care: From Evidence to Execution Authors: Joe O’Sullivan, PhD, MD, BAO, BCH, FRCR, FFRRCSI, FRCPI; Irene Burger, MD; Elena Castro, MD, PhD; Bertrand Tombal, MD, PhD

https://www.medscape.org/viewarticle/1003032?src=mkmcmr_driv_stan_mscpedu_260318-OUS-HONC-1003032-cta&uac=148436CN&sso=true

Mechanisms Underlying Hazardous Alcohol Use After Mild Traumatic Brain Injury

https://arcr.niaaa.nih.gov/volume/45/1/mechanisms-underlying-hazardous-alcohol-use-after-mild-traumatic-brain-injury?utm_source=arcr-email&utm_medium=email&utm_campaign=schindler-2026-03&utm_content=arcr-listserv Mechanisms Underlying Hazardous Alcohol Use After Mild Traumatic Brain Injury Makenzie Patarino, Jenna Sanders, and Abigail G. Schindler People who have experienced mild traumatic brain injuries (concussions)—for example, among military populations and contact-sport athletes—are at increased risk of alcohol misuse and alcohol use disorder. A new Alcohol Research: Current Reviews article explores biological mechanisms that may contribute to this increased risk, such as inflammation, degeneration of brain tissue, and altered brain and hormonal signaling. Understanding these associations may improve how people with mild traumatic brain injuries are treated to mitigate risk of alcohol misuse

Building Care Plans With Radiopharmaceuticals for Patients With mCRPC Authors: Neal D. Shore, MD, FACS; Alicia Morgans, MD, MPH, FASCO; Chad Tang, MD

https://www.medscape.org/viewarticle/building-care-plans-radiopharmaceuticals-patients-mcrpc-2026a10007zy?page=1&src=wnl_tpal_260318_mscpedu&sso=true&uac=148436CN&impID=8197864

Persistent Dysuria in a 30-Year-Old Man With Crohn’s Disease Aliza Green, MD; Paul E. Sax, MD March 17, 2026

https://reference.medscape.com/viewarticle/persistent-dysuria-30-year-old-man-crohns-disease-2026a10006v1?ecd=wnl_edit_tpal_etid8198738&uac=148436CN&impID=8198738

Immunotherapy for Non-Muscle-Invasive Bladder Cancer: Expert Takeaways From the Latest Updates Authors: Bernadett Szabados, MD; Maria De Santis, MD; Neal D. Shore, MD, FACS

https://www.medscape.org/viewarticle/immunotherapy-non-muscle-invasive-bladder-cancer-expert-2026a10005af?page=1&uac=148436CN&sso=true&src=mkmcmr_driv_stan_mscpedu_260318-OUS-HONC-immunotherapy-non-muscle-invasive-bladder-cancer-expert-2026a10005af-cta

When Psoriasis Txs Lose Their Punch | Biologics vs Phototherapy in Autoimmune Diseases +++++ ++++

TREATMENT ADVANCES When Psoriasis Treatments Lose Their Punch When Psoriasis Treatments Lose Their Punch: Understanding Tachyphylaxis Steven R. Feldman, MD, PhD https://www.medscape.com/viewarticle/when-psoriasis-treatments-lose-their-punch-understanding-2026a100076n?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 March 12, 2026 Biologics vs Phototherapy in Psoriasis: Cost-Benefit Analysis Biologics vs Phototherapy in Psoriasis: Cost-Benefit Analysis Highlights Divergent Patient and Payer Incentives John Jesitus https://www.medscape.com/viewarticle/biologics-vs-phototherapy-psoriasis-cost-benefit-analysis-2026a10007rd?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 March 13, 2026 New Autoimmune Disease CAR T Results ‘a Tremendous Success’ New Autoimmune Disease CAR T Results ‘a Tremendous Success’ Tara Haelle https://www.medscape.com/viewarticle/new-autoimmune-disease-car-t-results-tremendous-success-2026a10004ml?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 February 13, 2026 Biologic Approved for Adolescent Hidradenitis Suppurativa Secukinumab Gets FDA Nod for Adolescent Hidradenitis Suppurativa Heidi Splete https://www.medscape.com/viewarticle/secukinumab-gets-fda-nod-adolescent-hidradenitis-suppurativa-2026a10007wm?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 March 16, 2026 Alopecia Areata Tied to Higher Risk for Multiple Autoimmune Diseases Alopecia Areata Linked to Higher Risk for Multiple Autoimmune Diseases in Children, Females Edited by Gargi Mukherjee https://www.medscape.com/viewarticle/alopecia-areata-linked-higher-risk-multiple-autoimmune-2026a10006jt?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 March 03, 2026 AUTOIMMUNE DISEASE CARE Skin Inflammation Therapies Might Target Depression Skin Disease Immune Dysregulation Profile Shared by Depression Ted Bosworth https://www.medscape.com/viewarticle/skin-disease-immune-dysregulation-profile-shared-depression-2026a10007eh?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 March 11, 2026 FFA Appears Earlier in Patients With Skin of Color Frontal Fibrosing Alopecia Appears Earlier in Skin of Color Groups Edited by Sneha Gupta https://www.medscape.com/viewarticle/frontal-fibrosing-alopecia-appears-earlier-skin-color-groups-2026a100003j?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 January 02, 2026 Does Vitiligo Increase Risk for Mental Health Disorders? Does Vitiligo Increase Risk for Mental Health Disorders Compared to Other Skin Conditions? Edited by Vineeta Teotia https://www.medscape.com/viewarticle/does-vitiligo-increase-risk-mental-health-disorders-compared-2026a1000795?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 Managing Acute Psoriasis Flares: What to Know Fast Five Quiz: Acute Psoriasis Flares Reviewed by Alan Irvine, MD, DSc https://reference.medscape.com/viewarticle/acute-psoriasis-flares-2025a1000u6r?ecd=mkm_ret_260318_mscpmrk_derm_AutoimmuneSkinDisease_etid8197183&uac=148436CN&impID=8197183 December 22, 2025

How Are Oral SERDs Changing the Management of Advanced HR-Positive Breast Cancer? Authors: Erica L. Mayer, MD, MPH; François-Clément Bidard, MD, PhD

https://www.medscape.org/viewarticle/how-are-oral-serds-changing-management-advanced-hr-positive-2025a1000xkg?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260319-OUS-HONC-how-are-oral-serds-changing-management-advanced-hr-positive-2025a1000xkg-cta&sso=true

Même symptôme, deux réalités : ce que cache le prurit dans la Dermatite Atopique et le Prurigo Nodulaire

https://www.linkedin.com/pulse/m%C3%AAme-sympt%C3%B4me-deux-r%C3%A9alit%C3%A9s-ce-que-cache-le-prurit-dans-la-dermatite-ujtwe/ La dermatite atopique (DA) et le prurigo nodulaire (PN) sont deux maladies dermatologiques neuro-immunes chroniques distinctes qui partagent les démangeaisons comme symptôme le plus fréquent et le plus lourd. Le PN est une maladie rare avec une faible prévalence rapportée dans plusieurs pays comme les États-Unis (36,7 à 52,2 cas pour 100 000) et l’Allemagne (0,21 %). Les patients atteints de cette maladie peuvent présenter des démangeaisons impossibles à soulager et des lésions prurigineuses invalidantes. Les démangeaisons, l’inflammation, l’altération de la différenciation épidermique et la fibrose sont considérées comme les 4 caractéristiques du PN. En revanche, la DA est un trouble cutané courant qui touche 223 à 230 millions de personnes dans le monde. Les patients atteints de DA présentent des démangeaisons persistantes et des lésions cutanées eczémateuses. Démangeaisons, inflammation et perturbation de la barrière cutanée sont les 3 caractéristiques de la DA.

miércoles, 18 de marzo de 2026

Building Care Plans With Radiopharmaceuticals for Patients With mCRPC Authors: Neal D. Shore, MD, FACS; Alicia Morgans, MD, MPH, FASCO; Chad Tang, MD

https://www.medscape.org/viewarticle/building-care-plans-radiopharmaceuticals-patients-mcrpc-2026a10007zy?page=1

Older Adults With Advanced Cancer Prioritize Quality of Life Will Pass March 18, 2026

https://www.medscape.com/viewarticle/priorities-older-patients-cancer-anyone-listening-2026a10008ad

Tumor Debulking Fails to Boost Survival in Metastatic CRC Edited by Gargi Mukherjee March 18, 2026

https://www.medscape.com/viewarticle/tumor-debulking-fails-boost-survival-metastatic-crc-2026a100088m

New Adjuvant Option Emerges for Node-Positive ER+ Breast Cancer Kathy D. Miller, MD March 18, 2026

https://www.medscape.com/viewarticle/new-adjuvant-option-emerges-node-positive-er-breast-cancer-2026a100077x

A 2-Year-Old Girl With a Single Pink “Bump” on Her Abdomen Catalina Matiz, MD March 18, 2026

https://www.medscape.com/viewarticle/2-year-old-girl-single-pink-bump-her-abdomen-2026a10007d1

Early Imaging Could Improve Outcomes for Emergency Patients With Blood in Urine Becky McCall, PhD, MScPh March 16, 2026

https://www.medscape.com/viewarticle/early-imaging-could-improve-outcomes-emergency-patients-2026a1000816?ecd=wnl_edit_tpal_etid8195904&uac=148436CN&impID=8195904

Sleep your way to better metabolic health The Lancet Diabetes & Endocrinology ++... ++

Sleep your way to better metabolic health The Lancet Diabetes & Endocrinology https://www.thelancet.com/journals/landia/article/PIIS2213-8587(26)00047-1/fulltext?dgcid=raven_jbs_etoc_email Apr 2026 Volume 14Number 4p275-362, e7 https://www.thelancet.com/journals/landia/issue/vol14no4/PIIS2213-8587(26)X2002-2 Risk of recurrence after successful surgery for Cushing's disease and association with USP8 genotype and tumour size: an international, retrospective, longitudinal cohort study Qilin Zhang, PhDa,c,d,† ∙ Yixin Cai, PhDa,† ∙ Yuyu Liu, MDa,† ∙ Boyuan Yao, PhDa,† ∙ Prajina Sharmab ∙ Elisabeth Ruoffb ∙ et al. https://www.thelancet.com/journals/landia/article/PIIS2213-8587(25)00396-1/fulltext?dgcid=raven_jbs_etoc_feature_landia Tubeless automated insulin delivery versus multiple daily injections in children and adults with type 1 diabetes with elevated HbA1c (RADIANT): a multicentre, international, parallel-group, open-label, randomised, controlled trial Emma G Wilmot, PhDa,b ∙ Prof Jacques Beltrand, PhDc,d ∙ Prof Bruno Guerci, PhDe ∙ Aurelie Berot, MDf ∙ Prof Helene Hanaire, PhDg ∙ Elise Bismuth, MDh ∙ et al. https://www.thelancet.com/journals/landia/article/PIIS2213-8587(25)00364-X/abstract?dgcid=raven_jbs_etoc_feature_landia

Preventing organ transplant rejection

https://www.nih.gov/news-events/nih-research-matters/preventing-organ-transplant-rejection At a Glance Scientists found that the body’s lymphatic drainage system may play an unexpected role in chronic organ rejection. The findings point to potential new approaches for treating or preventing rejection of certain organ transplants.

Machine learning analysis of CT scans

https://www.nih.gov/news-events/nih-research-matters/machine-learning-analysis-ct-scans At a Glance Researchers developed a machine learning model that can interpret abdominal CT scans to make a diagnosis and possibly predict risk for some chronic diseases. The model could cut down on the time it takes to analyze and interpret CT scans.

Blood test predicts start of Alzheimer’s disease symptoms

https://www.nih.gov/news-events/nih-research-matters/blood-test-predicts-start-alzheimers-disease-symptoms At a Glance Researchers developed an Alzheimer’s “clock” that uses a blood test to roughly predict the onset of Alzheimer’s disease symptoms. The ability to predict symptom onset could aid in the selection of clinical trial participants and might one day enable improved decision-making for patients

Safer Stroke Prevention on the Horizon? What the New Factor XIa Inhibitor Data Reveal Authors: Eva A. Mistry, MBBS, MSCI, FAHA; Yi Dong, MD; Xin Cheng, MD, PhD; Mike Sharma, MD, MSc, FRCPC; Ashkan Shoamanesh, MD, PhD

https://www.medscape.org/viewarticle/safer-stroke-prevention-horizon-what-new-factor-xia-2026a10004zj?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260318-OUS-HONC-safer-stroke-prevention-horizon-what-new-factor-xia-2026a10004zj-cta&sso=true

NIH invests $150 million in human-based research to reduce use of animal models

NIH invests $150 million in human-based research to reduce use of animal models New program will develop, validate and standardize research tools to develop more sophisticated and relevant models of disease. https://www.nih.gov/news-events/news-releases/nih-invests-150-million-human-based-research-reduce-use-animal-models

Why mental health is a core part of the rare disease experience: listening to the patient voice

https://rarerevolutionmagazine.com/why-mental-health-is-a-core-part-of-the-rare-disease-experience-listening-to-the-patient-voice/ Often dealt with as an aside, mental health can be as significant as physical health, for both rare disease patients and caregivers. A recent survey by Rare Patient Voice highlights the effects of rare disease on mental health and the impact on all aspects of life when a patient and caregiver’s emotional wellbeing is not addressed and supported For people living with rare diseases, and for the family caregivers who support them, mental health is not separate from physical health. It is part of everyday life. It affects how people cope, how they make decisions and how they manage the long and often uncertain journey of rare disease.

ASCO Genitourinary Cancers Symposium 2026 February 26 - 28, 2026 San Francisco, California

Enfortumab Vedotin + Pembro Beats Cisplatin for Resectable MIBC M. Alexander Otto, PA, MMSc https://www.medscape.com/viewarticle/enfortumab-vedotin-pembro-beats-cisplatin-resectable-mibc-2026a10006gl?ecd=wnl_conf_onc_ASCO-GU-non-spon_260318_mscpedit_etid8194248&uac=148436CN&impID=8194248 March 02, 2026 ASCO Genitourinary Cancers Symposium 2026 February 26 - 28, 2026 San Francisco, California https://www.medscape.com/c25/p14/asco-genitourinary-cancers-symposium-2026-2026a100048v

Patient Stories: How Palliative Care Helps with Anxiety, Pain, and More

https://getpalliativecare.org/patient-stories-how-palliative-care-helps-with-anxiety-pain-and-more/?utm_source=Get+Palliative+Care&utm_campaign=db45426eae-GPC+-+Richard+Patient+Story+%2F+03.18.26&utm_medium=email&utm_term=0_4ef32a103e-db45426eae-378264050

Nanomedicines in the treatment of methicillin-resistant Staphylococcus aureus Anurag Yadav* [1] , Kusum Yadav [2]

https://www.academia.edu/academia-drug-development-and-pharmacotherapy/2/1/10.20935/AcadDrug8083 Abstract Methicillin-resistant Staphylococcus aureus (MRSA) has emerged as a formidable global health threat by evading conventional antibiotics and constraining therapeutic options. This review examines the clinical impasse and maps a rapidly expanding nanotechnological arsenal poised to overcome it. First, we delineate the molecular and pharmacological limitations of β-lactams, glycopeptides, and next-line agents, emphasizing the urgent need for orthogonal strategies. Central to this review are five converging nanodesign paradigms. Inorganic and metallic nanoconstructs, such as silver and gold colloids, magnetically responsive iron oxides, and near-infrared photothermal systems, exploit elemental properties to disrupt biofilms, catalyze reactive oxygen species, and induce localized hyperthermia. Mesoporous silica and graphene oxide-based platforms offer programmable porosity and surface tunability, allowing enzyme functionalization to degrade extracellular matrices and re-sensitize persister cells. Biomimetic and stimuli-responsive nanoparticles enhance targeting by cloaking in cellular membranes or releasing payloads in acidic, enzyme-rich MRSA environments. Hybrid constructs integrate antibiotics, photothermal triggers, or CRISPR-based gene editors to achieve synergistic effects while mitigating resistance evolution. Recognizing delivery bottlenecks, we highlight depot-forming hydrogels and thermoresponsive injectables that solidify in situ, maintaining high local drug concentrations within abscesses or infected bone. The review concludes by addressing translational challenges, including scalable synthesis, immunotoxicity, AI-guided formulation design, and regulatory alignment. Collectively, these advances signify a paradigm shift from empirical pharmacology to precision-engineered therapeutics, redefining MRSA as a tractable target amenable to multi-pronged clinical intervention. https://www.academia.edu/journals/academia-drug-development-and-pharmacotherapy/articles?source=journal-top-nav

2026 ACC/AHA/AACVPR/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Dyslipidemia: A Report of the American College of Cardiology/American Heart Association Joint Committee on Clinical Practice Guidelines

https://www.ahajournals.org/doi/10.1161/CIR.0000000000001423?utm_campaign=sciencenews25-26&utm_source=science-news&utm_medium=phd-link&utm_content=phd-3-13-26 2026 Guideline on the Management of Dyslipidemia Published: March 13, 2026 https://professional.heart.org/en/science-news/2026-guideline-on-the-management-of-dyslipidemia ACC/American Heart Association Issue Updated Guideline for Managing Lipids, Cholesterol https://newsroom.heart.org/news/accaha-issue-updated-guideline-for-managing-lipids-cholesterol What is Lipoprotein(a)? You’ll often see lipoprotein(a) referred to as Lp(a), pronounced “L-P-little-A.” https://familyheart.org/high-lipoprotein-a New Family Heart study: Increased lipoprotein(a) levels continuously increase risk for recurrent cardiovascular events https://familyheart.org/lpa-recurrent-ascvd-events-2025 LEAD Pediatric Cholesterol Screening Initiative Addressing the Lack of Universal Pediatric Cholesterol Screening in the United States https://familyheart.org/lead-pediatric-cholesterol-screening-initiative Predicting Risk of cardiovascular disease EVENTs (American Heart Association PREVENT™) https://professional.heart.org/en/guidelines-and-statements/about-prevent-calculator

Beyond PSMA: Preparing Your Practice for the Next Wave of Prostate Cancer Biomarkers Authors: Karim Fizazi, MD, PhD; Bertrand Tombal, MD, PhD; Alberto Briganti, MD, PhD

https://www.medscape.org/viewarticle/beyond-psma-preparing-your-practice-next-wave-prostate-2026a10006ql?page=1&sso=true&uac=148436CN&src=mkmcmr_reeng_recap_mscpedu_activity thank you for your recent participation in the activity: Beyond PSMA: Preparing Your Practice for the Next Wave of Prostate Cancer Biomarkers Below are some key learning points to help reinforce the impact of this activity. ☑ Current guidelines (EAU/NCCN) recommend early somatic testing for HRR mutations and MSI/dMMR in all metastatic patients, preferably before first-line mCRPC treatment. Testing should be performed on metastatic tissue when feasible, with primary tumor or ctDNA as alternatives when biopsy is not possible. ☑ Several biomarkers have demonstrated predictive value in clinical trials: PTEN loss predicts benefit from capivasertib + abiraterone (CAPItello-281 trial), HRR mutations (especially BRCA1/2) predict response to PARP inhibitors (AMPLITUDE trial among others), and MSI-H/dMMR status potentially requires immunotherapy treatment. ☑ Best practices include biomarker assessment with repeat PSMA PET imaging at each treatment change point and consideration of retesting with new tissue biopsy or ctDNA to capture tumor evolution. High-risk localized disease warrants early molecular characterization via IHC and NGS. ☑ Multidisciplinary approach is essential for optimal biomarker utilization, involving urologists, medical oncologists, pathologists, radiologists, genetic counselors, nurses, physiotherapists, among others to properly interpret results and implement personalized treatment strategies based on the molecular profile.

Current and Future Oral SERD Strategies in the Management of ER-Positive/HER2-Negative MBC: Analyzing the Clinical Data Authors: Komal Jhaveri, MD, FACP, FASCO; Shaheenah Dawood, MD, MPH

https://www.medscape.org/viewarticle/current-and-future-oral-serd-strategies-management-er-2026a100022b?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260317-OUS-HONC-current-and-future-oral-serd-strategies-management-er-2026a100022b-cta&sso=true

Medscape Now! Huntington Disease: An Overview for the Interprofessional Care Team Authors: Blessing Ajayi, PA-C

https://www.medscape.org/viewarticle/medscape-now-huntington-disease-overview-interprofessional-2026a10007gg?page=1&impID=8195042&src=wnl_tpal_260317_mscpedu&uac=148436CN&sso=true

martes, 17 de marzo de 2026

European Association of Urology (EAU) 2026 Annual Meeting

https://www.medscape.com/c25/p14/european-association-urology-eau-2026-annual-meeting-2026a10005ne

Postpolypectomy Surveillance: Where Guidelines Clash Álvaro de la Serna, MD March 17, 2026

https://www.medscape.com/viewarticle/postpolypectomy-surveillance-where-guidelines-clash-2026a100082e

Treatment Delays in Colorectal Cancer More Common in Urban Men and Racial Minorities Edited by Gargi Mukherjee March 17, 2026

https://www.medscape.com/viewarticle/treatment-delays-colorectal-cancer-more-common-urban-men-and-2026a1000856

Adjuvant Systemic Therapy Linked to Improved Long-Term Survival Outcomes in Patients With Bilateral Breast Cancer Edited by Vineeta Teotia Medscape UK March 16, 2026

https://www.medscape.com/viewarticle/adjuvant-systemic-therapy-linked-improved-long-term-survival-2026a10007ln

Study Identifies Common Occupational Allergens Affecting Healthcare Workers With Contact Dermatitis Edited by Gargi Mukherjee March 17, 2026

https://www.medscape.com/viewarticle/study-identifies-common-occupational-allergens-affecting-2026a1000826

GLP-1 Use Linked to Lower Mortality in Patients With Brain Metastases Edited by Victoria Stern March 17, 2026

https://www.medscape.com/viewarticle/glp-1-usame-linked-lower-mortality-patients-brain-metastases-2026a1000848

What Does FDA Food Pyramid Mean for Cancer Risk and Care? Tara Haelle March 16, 2026

https://www.medscape.com/viewarticle/what-does-fda-food-pyramid-mean-cancer-risk-and-care-2026a10007xc

Precision Oncology in HER2-Mutated NSCLC: Strategies for Today and Tomorrow Authors: Yong Fang, MD; Hye Ryun Kim, MD, PhD; Hibiki Udagawa, MD, PhD

https://www.medscape.org/viewarticle/precision-oncology-her2-mutated-nsclc-strategies-today-and-2025a100105r?page=1&sso=true&src=mkmcmr_driv_stan_mscpedu_260317-OUS-HONC-precision-oncology-her2-mutated-nsclc-strategies-today-and-2025a100105r-cta&uac=148436CN

New risk models improve food safety guidelines for pregnant individuals

https://www.news-medical.net/news/20260317/New-risk-models-improve-food-safety-guidelines-for-pregnant-individuals.aspx Listeria is the third-leading cause of death among bacterial foodborne pathogens in the U.S. and pregnant individuals bear a disproportionate share of that burden. Yet the scientific models used to set food safety policy have rarely been designed with pregnant people specifically in mind. A new study to be published in Risk Analysis aims to change that.

Viewpoint: RFK, Jr.’s unethical plan to run a Tuskegee-like syphilis experiment in West Africa to ‘prove’ hepatitis B vaccines cause neurological damage Pete Shanks | March 17, 2026

https://geneticliteracyproject.org/2026/03/17/viewpoint-rfk-jr-s-unethical-plan-to-run-a-tuskegee-like-syphilis-experiment-in-west-africa-to-prove-hepatitis-b-vaccines-cause-neurological-damage/

U.S. defunding International Agency for Research on Cancer (IARC), UN-affiliated group that issued cancer warnings for glyphosate, steak, and getting a haircut Jocelyn Kaiser | Science | March 17, 2026

https://geneticliteracyproject.org/2026/03/17/u-s-defunding-international-agency-for-research-on-cancer-iarc-un-affilated-group-that-issued-cancer-warnings-for-glyphosate-steak-and-getting-a-haircut/

Clinical trial results support use of weekly extended-release buprenorphine for treatment of opioid use disorder during pregnancy NIH-supported study shows this treatment resulted in higher rates of illicit opioid abstinence than current standard of care.

Clinical trial results support use of weekly extended-release buprenorphine for treatment of opioid use disorder during pregnancy NIH-supported study shows this treatment resulted in higher rates of illicit opioid abstinence than current standard of care. https://www.nih.gov/news-events/news-releases/clinical-trial-results-support-use-weekly-extended-release-buprenorphine-treatment-opioid-use-disorder-during-pregnancy The Helping to End Addiction Long-term® Initiative The NIH HEAL Initiative® is an NIH-wide effort to improve prevention and treatment strategies for opioid misuse and addiction and to enhance pain management.​ https://www.nih.gov/heal

RISE Together: Data Sharing Across the Rare Disease Ecosystem Fecha y hora 30 mar 2026 10:00 a. m.

https://duke.zoom.us/webinar/register/WN_OJ2IqzitQWuv7WKqRQZC3g?utm_medium=email&utm_source=govdelivery#/registration RISE Together: Data Sharing Across the Rare Disease Ecosystem Date: Monday, March 30, 2026; 9 a.m. - 4:30 p.m. ET This public workshop, co-convened by the Duke-Margolis Institute for Health Policy and the FDA Rare Disease Innovation Hub, is designed for all stakeholders in the rare disease community to explore data sharing as it pertains to informing development and regulatory review for rare disease therapies. The workshop will focus on clarifying possible avenues for data sharing and the types of data that can be shared (e.g., safety information, real-world evidence, and deidentified patient data). The workshop will also discuss: the promotion of data sharing practices and structures for facilitating data sharing among rare disease drug development stakeholders; examples of the impact of data sharing in regulatory submissions; a priori considerations for the collection and sharing of high quality data; some of the logistical and legal challenges encountered in data sharing; and whether there are ways that FDA might support data sharing, within the bounds of its authority.

Exploring the Scope of Dietary Supplement Ingredients(Virtual Registration) Online event Friday, Mar 27 from 10 am to 4 pm GMT-3

Exploring the Scope of Dietary Supplement Ingredients(Virtual Registration) Online event Friday, Mar 27 from 10 am to 4 pm GMT-3 https://www.eventbrite.com/e/exploring-the-scope-of-dietary-supplement-ingredientsvirtual-registration-tickets-1983904155735?aff=oddtdtcreator&utm_medium=email&utm_source=govdelivery Public Meeting Exploring the Scope of Dietary Supplement Ingredients Date: Friday, March 27, 2026; 9 a.m. - 3 p.m. ET FDA’s Office of Dietary Supplement Programs (ODSP) invites stakeholders to participate in a public meeting to discuss the evolving landscape of dietary supplement ingredients and how recent scientific and technological advances are shaping the industry.

ASCO Genitourinary Cancers Symposium 2026

https://www.medscape.com/c25/p14/asco-genitourinary-cancers-symposium-2026-2026a100048v?ecd=wnl_conf_onc_ASCO-GU-non-spon_260316_mscpedit_etid8185396&uac=148436CN&impID=8185396

Highlights From the Annual Neuro-Oncology Conference: Integrating IDH Inhibitor Therapy Into Glioma Care Authors: Maciej Mrugala, MD, PhD, MPH; Rimas V. Lukas, MD; Yoshie Umemura, MD

https://www.medscape.org/viewarticle/1003175?uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260317-OUS-HONC-1003175-cta&sso=true

RARE Rev-inar episode 024: Neurofibromatosis type 1 (NF1): navigating diagnosis, care, and transition Fecha y hora 19 mar 2026 11:00 a. m.

https://us02web.zoom.us/webinar/register/WN_nY8AFTRxQtePkbt4QNkBHw#/registration Don't miss our conversation on Neurofibromatosis type 1 (NF1)! Hear from paediatric CNS Katrina Kettle on the function of the new NF1 passport and essential early independence-building strategies to ensure a smooth transition to adult services. Book your free place! https://editions.rarerevolutionmagazine.com/html5/reader/production/default.aspx?pubname=&edid=b226070d-f1dd-4177-bfbc-65a7f9ba2588&pnum=40 Advocate, mother and physician Dr Mariëlle van den Berg shares her personal and professional journey at the forefront of Rett Syndrome progress. Highlighting results from a recent white paper and international Burden of Illness survey, she reveals the hidden mental, financial and practical struggles families face. With bold recommendations for policy reform in lifelong care, Mariëlle calls for Europe-wide standards and empowers families to use these findings to secure better support at every stage.

'Concerning': CRC Continues to Shift Toward Younger Adults Megan Brooks March 15, 2026

https://www.medscape.com/viewarticle/concerning-crc-continues-shift-toward-younger-adults-2026a10007w2?ecd=wnl_edit_tpal_etid8193136&uac=148436CN&impID=8193136

Erectile Dysfunction in Young Man Reveals Prolactinoma Thomas Kron, MD Medscape Europe March 16, 2026

https://www.medscape.com/viewarticle/erectile-dysfunction-young-man-reveals-prolactinoma-2026a10007xf?ecd=wnl_tp10_daily_260316_MSCPEDIT_etid8191410&uac=148436CN&impID=8191410

How much do GLP-1s reduce overdoses and substance-related deaths in patients with prior substance use disorders? (Medscape AI)

https://www.medscape.com/ai-search?query=How%20much%20do%20GLP-1s%20reduce%20overdoses%20and%20substance-related%20deaths%20in%20patients%20with%20prior%20substance%20use%20disorders%3f&ecd=wnl_tp10_daily_260316_MSCPEDIT_etid8191410&uac=148436CN&impID=8191410

lunes, 16 de marzo de 2026

Doctors treating sickle cell disease report the highest burnout rates

https://www.news-medical.net/news/20260316/Doctors-treating-sickle-cell-disease-report-the-highest-burnout-rates.aspx Physicians who treat sickle cell disease face some of the most complex and resource-intensive care in medicine, and a national survey now shows they experience markedly higher burnout, raising concerns about workforce sustainability and patient care.

Scientists show gut bacteria can reach the brain in mice and reveal a potential vagus nerve pathway

https://www.news-medical.net/news/20260315/Scientists-show-gut-bacteria-can-reach-the-brain-in-mice-and-reveal-a-potential-vagus-nerve-pathway.aspx A new mouse study reveals that under certain dietary conditions, gut bacteria can reach the brain without entering the bloodstream, suggesting a possible vagus nerve route and raising new questions about the gut–brain connection.

PTEN Deficiency in Prostate Cancer: Biology, Prognosis, and Therapeutic Relevance Authors: Elena Castro, MD, PhD

https://www.medscape.org/viewarticle/pten-deficiency-prostate-cancer-biology-prognosis-and-2025a1000rqh?page=1&src=mkmcmr_driv_stan_mscpedu_260316-OUS-HONC-pten-deficiency-prostate-cancer-biology-prognosis-and-2025a1000rqh-cta&uac=148436CN&sso=true

Cutaneous T-Cell Lymphoma by CheckRare Staff|

https://checkrare.com/cutaneous-t-cell-lymphoma-2/

Hemophilia Clinical Research Highlights: ISTH 2025

https://checkrare.com/learning/p-isth2025-module1-hemophilia-clinical-research-highlights/

Immune Thrombocytopenia Clinical Research Highlights: ISTH 2025

https://checkrare.com/learning/p-isth2025-module2-immune-thrombocytopenia-clinical-research-highlights/

Immune Thrombotic Thrombocytopenic Purpura (iTTP) Clinical Research Highlights: ISTH 2025

https://checkrare.com/learning/p-isth2025-module3-immune-thrombotic-thrombocytopenic-purpura-ittp-clinical-research-highlights/

Potential Treatment for ROSAH Syndrome Accepted into FDA’s Rare Disease Evidence Principles Process Mar 12, 2026 | Autoimmune / Autoinflammatory Disorders +...

https://checkrare.com/ The US Food and Drug Administration (FDA) has accepted DF-003 into the FDA Rare Disease Evidence Principles Process (RDEP) for the potential treatment of ROSAH syndrome. ROSAH (retinal dystrophy,... AND MORE!!!

CLINICAL PERSPECTIVES +++++ +++++ +++++ +++

CLINICAL PERSPECTIVES MajesTEC-9 Clinical Trial of Teclistamab in Patients With Multiple Myeloma https://checkrare.com/majestec-9-clinical-trial-of-teclistamab-in-patients-with-multiple-myeloma/ Roberto Mina, MD, Associate Professor at Winship Cancer Institute of Emory University, discusses the Majestec-9 clinical trial of Tecvayli (teclistamab) in patients with multiple myeloma (MM). Long-Term Outcomes From the PIONEER Study in Patients With Indolent Systemic Mastocytosis https://checkrare.com/long-term-outcomes-from-the-pioneer-study-in-patients-with-indolent-systemic-mastocytosis/ Tsewang Tashi, MD, Hematologist at the Huntsman Cancer Institute at the University of Utah, discusses long-term data from the PIONEER clinical trial examining the use of avapritinib in patients with indolent systemic mastocytosis (ISM). Using Artificial Intelligence to Analyze Castleman Disease Histopathology https://checkrare.com/using-artificial-intelligence-to-analyze-castleman-disease-histopathology/ Robert S. Ohgami, MD, PhD, Professor of Pathology, University of Utah, and Founding Vice President and Chief Medical Director, ARUP Institute for Research and Innovation, discusses the utilization of artificial intelligence (AI) to analyze Castleman disease (CD) histopathology. Elritercept’s Effect on Transfusion Independence in Patients With Myelodysplastic Syndromes https://checkrare.com/elritercepts-effect-on-transfusion-independence-in-patients-with-myelodysplastic-syndromes/ Lynette Chee, PhD, Hematologist at The Royal Melbourne Hospital/ Peter MacCallum Cancer Centre, discusses elritercept’s effect on transfusion independence (TI) in patients with myelodysplastic syndromes (MDS). 52-Week Results From the VERIFY Clinical Trial of Rusfertide in Patients With Polycythemia Vera https://checkrare.com/52-week-results-from-the-verify-clinical-trial-of-rusfertide-in-patients-with-polycythemia-vera/ Andrew T. Kuykendall, MD, VERIFY Lead Investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center, discusses 52-week results from the VERIFY clinical trial testing rusfertide in patients with polycythemia vera (PV). Results from the XTEND-ed Clinical Trial Evaluating Efanesoctocog Alfa for the Treatment of Patients With Hemophilia A https://checkrare.com/results-from-the-xtend-ed-clinical-trial-evaluating-efanesoctocog-alfa-for-the-treatment-of-patients-with-hemophilia-a/ Lynn Malec, MD, Versiti Blood Research Institute and Medical College of Wisconsin, discusses results from the XTEND-ed clinical trial evaluating efanesoctocog alfa for the treatment of patients with hemophilia A. Bleximenib Combination Therapy for Patients With Acute Myeloid Leukemia https://checkrare.com/bleximenib-combination-therapy-for-patients-with-acute-myeloid-leukemia/ Hartmut Döhner, MD, Professor of Medicine and Medical Director of the Department of Hematology and Oncology, Ulm University, Germany, discusses bleximenib combination therapy for treatment of patients with acute myeloid leukemia (AML). Rare Diseases in Ireland – New Efforts to Improve Access to Care https://checkrare.com/rare-diseases-in-ireland-new-efforts-to-improve-access-to-care/ Each country takes a different approach to rare diseases, from the way it defines the term to the health policies it implements to its approach to research. SWITCH Clinical Trial Design for Patients With Hemophilia A https://checkrare.com/switch-clinical-trial-design-for-patients-with-hemophilia-a/ Guy Young, MD, Children’s Hospital Los Angeles, discusses the SWITCH clinical trial design for patients with hemophilia A. Phase 2 Results of Dusquetide in the Treatment of Oral Ulcers Caused by Behçet’s Disease https://checkrare.com/phase-2-results-of-dusquetide-in-the-treatment-of-oral-ulcers-caused-by-behcets-disease/ Recently, results from a phase 2a study evaluating SGX945 (dusquetide) for the treatment of Behçet’s disease were published in Rheumatology (Oxford). Real World Safety Data of Enjaymo (Sutimlimab) in Patients With Cold Agglutinin Disease https://checkrare.com/real-world-safety-data-of-enjaymo-sutimlimab-in-patients-with-cold-agglutinin-disease/ Catherine M. Broome, MD, Professor of Medicine and Director of Cellular Apheresis, Lombardi Cancer Center, MedStar Georgetown University, discusses real world safety data of Enjaymo (sutimlimab) in patients with cold agglutinin disease (CAD). Long-term Efficacy of Rilzabrutinib in Patients With Immune Thrombocytopenia https://checkrare.com/long-term-efficacy-of-rilzabrutinib-in-patients-with-immune-thrombocytopenia/ David Kuter, MD, DPhil, Professor of Medicine at Harvard Medical School, Massachusetts General Hospital, discusses treatment with rilzabrutinib in patients with immune thrombocytopenia (ITP). Recent Data on Ravulizumab in Rare Hematologic Conditions https://checkrare.com/recent-data-on-raulizumab-in-rare-hematologic-disorders/ Anita Hill, MD, PhD, Global Medical Head for Hematology & Nephrology and Transplant at Alexion, AstraZeneca Rare Disease, discusses recent data on ravulizumab in rare hematologic conditions. Efficacy of Pozelimab + Cemdisiran To Treat Paroxysmal Nocturnal Hemoglobinuria https://checkrare.com/efficacy-of-pozelimab-cemdisiran-to-treat-paroxysmal-nocturnal-hemoglobinuria/ Jun Ho Jang, MD, PhD, Professor, Division of Hematology-Oncology at Samsung Medical Center, discusses results from an open-label extension study testing pozelimab plus cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH), who previously received the combination therapy or received ravulizumab. Treatment With Elritercept for Patients With Myelofibrosis https://checkrare.com/treatment-with-elritercept-for-patients-with-myelofibrosis/ Ciro Rinaldi, MD, Consultant Hematologist and Professor of Hematology at United Lincolnshire Hospital, discusses treatment with elritercept for patients with myelofibrosis (MF). Updated Phase 2 Results of Telquetamab + Teclistamab in Multiple Myeloma https://checkrare.com/updated-phase-2-results-of-telquetamab-teclistamab-in-multiple-myeloma/ Saad Usmani, MD, Myeloma Specialist and Cellular Therapist at Memorial Sloan Kettering Cancer Center, discusses updated results from the phase 2 RedirecTT-1 study of telquetamab + teclistamab in patients with multiple myeloma (MM). Results from the CARTITUDE-4 Clinical Trial in Multiple Myeloma https://checkrare.com/results-from-the-cartitude-4-clinical-trial-in-multiple-myeloma/ Binod Dhakal, MD, Associate Professor of Medicine at Medical College of Wisconsin, discusses results from the CARTITUDE-4 clinical trial in patients with multiple myeloma (MM). Results of Part 1a of the OLYMPIA-3 Study of Odronextamab+Chemotherapy in Diffuse Large B Cell Lymphoma https://checkrare.com/results-of-part-1a-of-the-olympia-3-study-of-odronextamabchemotherapy-in-diffuse-large-b-cell-lymphoma/ Jean-Marie Michot, MD, Institut Gustave Roussy, France, discusses results from Part 1A (dose escalation) of the OLYMPIA-3 study of odronextamab plus chemotherapy in patients with diffuse large B-cell lymphoma (DLBCL).

2026 Orphan Drugs: PDUFA Dates and FDA Approvals ++++++

FDA NEWS 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2026. Accelerated Approval Granted to Loargys (pegzilarginase) for the Treatment of ARG1 Deficiency https://checkrare.com/accelerated-approval-granted-to-loargys-pegzilarginase-for-the-treatment-of-arg1-deficiency/ The US FDA has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of patients ages 2 years and older with arginase 1 deficiency (ARG1-D). FDA Approves Venetoclax Combination Therapy for Adults With Chronic Lymphocytic Leukemia https://checkrare.com/fda-approves-venetoclax-combination-therapy-for-adults-with-chronic-lymphocytic-leukemia/ The US FDA has approved Venclexta (venetoclax) in combination with acalabrutinib therapy for untreated adult patients with chronic lymphocytic leukemia (CLL). FDA Approved Darzalex Faspro Combination Therapy for Patients With Transplant Ineligible Newly Diagnosed Multiple Myeloma https://checkrare.com/fda-approved-darzalex-faspro-combination-therapy-for-patients-with-transplant-ineligible-newly-diagnosed-multiple-myeloma/ The US FDA has approved Darzalex Faspro (daratumumab and hyaluronidase) in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for the treatment of adults with newly diagnosed multiple myeloma (MM) who are ineligible for autologous stem cell transplant (ASCT). Expanded Indication for Cablivi to Patients Ages 12 Years and Older With aTTP https://checkrare.com/fda-expands-cablivi-caplacizumab-indication-to-patients-ages-12-years-and-older-with-attp/ The U.S. FDA has approved Cablivi (caplacizumab) for the treatment of pediatric patients ages 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. FDA Approves Aqvesme (Mitapivat) for Treating Anemia in Alpha- and Beta-Thalassemia https://checkrare.com/fda-approves-aqvesme-mitapivat-for-treating-anemia-in-alpha-and-beta-thalassemia/ The U.S. FDA has approved Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.

Survey Finds Wide-Ranging Impact of X-Linked Hypophosphatemia (XLH) on Patient Experiences

https://checkrare.com/survey-finds-wide-ranging-impact-of-x-linked-hypophosphatemia-on-patient-experiences/

Position-wise mutation analysis and temporal changes in SARS CoV-2 Envelope (E) protein variants Anwesa Saha [1,†] , Diganta Mukherjee [1,†] , Aparna Mukhopadhyay* [1]

https://www.academia.edu/3064-9765/2/4/10.20935/AcadMolBioGen8011 Abstract SARS CoV-2 is a positive-sense, single-stranded RNA virus. The genome of the virus undergoes numerous mutations, making the development of universally effective drugs challenging. Among its structural proteins, the Envelope (E) protein acts as an ion transporter and virulence factor, making it a potential therapeutic target. Based on the literature available to date, we have identified several functionally important sites in the E protein. These include residues involved in lysosomal deacidification; those of the FYXY motif, involved in amyloid formation in the host; and the PDZ-binding DLLV motif. We focus our analysis on the significance of these residues while also searching for other interesting mutational patterns. In this study, we conducted a comprehensive mutational analysis of the SARS CoV-2 E protein utilizing bioinformatics, statistics, and structural modeling tools. Over 1.4 million sequences were retrieved from the NCBI virus database, filtered, clustered, and aligned chronologically. By employing a combination of web-based tools and in-house Python scripts, we analyzed per-residue Shannon entropy, mutation types, evolutionary pressure, and predicted structural impact (via ∆∆G). We observed a significant number of residues under diversifying selection. This suggests that new amino acids are being sampled at various positions in the protein, providing functional or structural benefits to the virus. A cyclical pattern of mutation and reversion was observed at position 9, stabilizing at a particular mutation. Similar trends appeared at position 11. These mutations may be functionally relevant, which need to be explored in future. However, the key regions have remained conserved over time. https://www.academia.edu/journals/academia-molecular-biology-and-genomics/articles?source=journal-top-nav

Pediatric Low-Grade Glioma With BRAF Alterations: Stories, Science, and Strategies Authors: Darren Hargrave, MBChB, MD; Cameron Miller; Enrico Opocher, MD; Sébastien Perreault, MD, FRCPC

https://www.medscape.org/viewarticle/pediatric-low-grade-glioma-braf-alterations-stories-science-2025a1000x3w?page=1&uac=148436CN&src=mkmcmr_driv_stan_mscpedu_260315-OUS-HONC-pediatric-low-grade-glioma-braf-alterations-stories-science-2025a1000x3w-cta&sso=true

Leadership skills training needs of early career doctors: a European survey Mário J. Santos, Ana Samouco, Zetti Azvee, Deepa Bagepalli Krishnan, Nikola Žaja, João Costa Pedro, Raka Maitra, Alma László, Beatriz Jorge, Matthäus Fellinger, Filipe Grilo, Mariana Pinto da Costa, Uriel Halbreich Volume 3, Issue 1

https://www.academia.edu/2997-9196/3/1/10.20935/MHealthWellB8187 Abstract Introduction: This study aims to investigate the access to leadership development opportunities among European psychiatric trainees and early career psychiatrists (ECPs) and their perceptions and needs related to leadership skills training. Materials and methods: This is a cross-sectional study using an online survey. The collected data was statistically analyzed using Stata 13, including an ordered probit regression to study the impact of five demographic characteristics of participants on the prioritization of 18 variables of leadership skills and 12 variables of management skills. Results: A total of 119 psychiatry trainees from 31 European countries responded to the study, of which 80 (67.2%) were female. Additionally, 48.7% of participants (n = 58) were general adult psychiatric trainees, and 29.4% (n = 35) were ECPs. Our results show that 63.0% (n = 75) had no access to leadership skills training within their training program. Also, respondents tended to be unsatisfied with the training received. More than half (n = 62, 52.1%) of trainees sought additional leadership and management training outside their program. All the participants recommended that training in leadership skills should be included in a psychiatric training program. The top three most important skills to trainees were “communication”, “teamwork”, and “empathy and cultural sensitivity” regarding leadership skills; and “stress management”, “time management”, and “crisis management” concerning management skills. Conclusions: Our study provides an overview of important gaps in the need, availability, and access to leadership skills training amongst psychiatric trainees and ECPs across Europe. We hope this study will help inform future developments and improvements in leadership skills training for trainees and ECPs across Europe. https://www.academia.edu/journals/academia-mental-health-and-well-being/articles?source=journal-top-nav

A 56-Year-Old With Shoulder Pain, Ear Edema, and Facial Rash Diana M. Girnita, MD, PhD January 20, 2026

https://reference.medscape.com/viewarticle/56-year-old-shoulder-pain-ear-edema-and-facial-rash-2026a10000o3?ecd=WNL_casechlg_260315_MSCPREF_etid8184808&uac=148436CN&impID=8184808

Recurrent Near-Fainting Episodes in a 62-Year-Old Construction Supervisor With Asthma and Back Pain Heidi Moawad, MD January 26, 2026

https://reference.medscape.com/viewarticle/recurrent-near-fainting-episodes-62-year-old-construction-2026a10001kj?ecd=WNL_casechlg_260315_MSCPREF_etid8184808&uac=148436CN&impID=8184808

Spontaneous Bruising and Vaginal Bleeding in a Middle-Aged Woman Belén Morales Franco, MD February 09, 2026

https://reference.medscape.com/viewarticle/spontaneous-bruising-and-vaginal-bleeding-middle-aged-woman-2026a10003md?ecd=WNL_casechlg_260315_MSCPREF_etid8184808&uac=148436CN&impID=8184808

A Nonsmoker With a Persistent Stubborn Cough Juan Antequera Martin-Portugués, DO February 20, 2026

https://reference.medscape.com/viewarticle/nonsmoker-persistent-stubborn-cough-2026a10004fp?ecd=WNL_casechlg_260315_MSCPREF_etid8184808&uac=148436CN&impID=8184808

Navigating Advances in TRK Fusion-Positive Cancers: A Collaborative Journey From Testing to Therapy Authors: David S. Hong, MD; Theodore W. Laetsch, MD; Jonathan C. Trent, MD, PhD

https://www.medscape.org/viewarticle/navigating-advances-trk-fusion-positive-cancers-2025a1000z5i?page=1&src=mkmcmr_driv_stan_mscpedu_260315-OUS-HONC-navigating-advances-trk-fusion-positive-cancers-2025a1000z5i-cta&uac=148436CN&sso=true

NF1-Associated Plexiform Neurofibromas in Adults: Which Patients Could Benefit From Emerging Systemic Therapies? Authors: Andrea Gross, MD; João Passos, MD

https://www.medscape.org/viewarticle/1003044?src=mkmcmr_driv_stan_mscpedu_260316-OUS-HONC-1003044-cta&sso=true&uac=148436CN

domingo, 15 de marzo de 2026

Gut microbes help turn white fat into calorie-burning beige fat A new study shows how specific gut bacteria work with diet to trigger fat browning in mice, revealing potential pathways for future metabolic therapies.

https://www.drugdiscoverynews.com/gut-microbes-help-turn-white-fat-into-calorie-burning-beige-fat-17065 When we think about fat, we most often imagine white fat cells, which store excess calories like tiny batteries, providing insulation and cushioning for muscles, bones, and organs. But not all fat is built to store energy. Brown fat does the opposite, burning calories to generate heat and keep the body warm. And then there is beige fat. First identified in 2008, beige fat resides within white fat tissue but functions like brown fat, efficiently burning energy when the body is exposed to cold.

Weekly Rundown: FDA approves first treatment for rare cerebral folate deficiency Unexpected breakthroughs in drug manufacturing, lawsuits, regulatory updates, and more led the news this week.

https://www.drugdiscoverynews.com/weekly-rundown-fda-approves-first-treatment-for-rare-cerebral-folate-deficiency-17069 The FDA has approved the first treatment for cerebral folate transport deficiency, a rare genetic disorder that impairs folate transport into the brain. Wellcovorin (leucovorin calcium) tablets have traditionally been used to reduce symptoms from high-dose chemotherapy but are now approved for adult and pediatric patients with a confirmed FOLR1 (folate receptor alpha 1) gene variant. This provides the first FDA-sanctioned option for a condition that can cause severe developmental delays, seizures, movement disorders, and other neurological complications. The approval was supported by a systematic review of 26 published case reports and case reviews from 2009 to 2024, covering 46 patients treated through various administration routes. Among 27 patients receiving oral leucovorin, 24 showed clinical improvements, including reduced seizure severity, better motor function, and enhanced communication or behavior. FDA Commissioner Marty Makary called the decision a “significant milestone” for patients who previously had no approved therapies, while officials noted that observational and real-world evidence played a key role in demonstrating clinical benefit. – Bree Foster

Microbes make, stabilize, and test drugs all inside a cell A new bacterial platform can produce millions of stabilized peptides in a single experiment, accelerating the search for therapies against hard-to-drug targets.

https://www.drugdiscoverynews.com/microbes-make-stabilize-and-test-drugs-all-inside-a-cell-17060?utm_campaign=DDN_Newsletter_Science%20Spotlight&utm_medium=email&_hsenc=p2ANqtz--HnIU43LWTvQmvUrjnwI-Jts60wFM0-Rhu70sSeiTydtR19hwi6EFPj5tR6iVGwP74EYmeJw-WZ20Flp2qw2nWY8yNSQ&_hsmi=408663516&utm_content=408663516&utm_source=hs_email Peptide therapeutics are experiencing a surge of interest, driven by significant advances, including the blockbuster success of weight loss drugs like semaglutide and tirzepatide. With more than 80 peptide drugs already on the market and hundreds more in clinical and preclinical development, the global market for peptide-based therapeutics is expected to grow to $68.83 billion by 2028.

Editorial Amplifying African voices to lead science for better health The Lancet Regional Health – Africa +...

https://www.thelancet.com/issue/S3050-5011(26)X2001-6

MRI-based personalisation of neoadjuvant chemotherapy duration in HER2-positive early breast cancer (TRAIN-3): primary results from a multicentre, single-arm, phase 2 study

Articles MRI-based personalisation of neoadjuvant chemotherapy duration in HER2-positive early breast cancer (TRAIN-3): primary results from a multicentre, single-arm, phase 2 study +... Fleur M LouisAnna van der VoortMette S van RamshorstAntonios DaletzakisIngrid A MandjesInge Kemperand others The Lancet OncologyVol. 27No. 3p349–360 https://www.thelancet.com/collections/tumour-imaging?dgcid=hubspot_email_infocusalerts-oncology_feature&parent=001611&startPage=&utm_campaign=infocusalerts-oncology&utm_medium=email&_hsenc=p2ANqtz-8zORBPB5oOMjMGjJRFxmbfxCo4WHwqNEwUXxyQ-eYVpxP02ggQAvFUEw3VnO-p4q2Pdadgfwsl8UVZYhcs9ozBF5z-1w&_hsmi=408798171&utm_content=408446946&utm_source=hs_email Published: February 16, 2026

CURE ID App Lets Clinicians Report Novel Uses of Existing Drugs

https://www.fda.gov/drugs/science-and-research-drugs/cure-id-app-lets-clinicians-report-novel-uses-existing-drugs?utm_medium=email&utm_source=govdelivery On March 15th, International Long COVID Awareness Day, use your experience to guide clinical trials and help countless others suffering with Long COVID. CURE ID is a treatment registry where patients, care partners, and clinicians unite to share what works, as well as what doesn’t. By using CURE ID, you can https://cure.ncats.io/pilot-overview/long-covid?utm_medium=email&utm_source=govdelivery Share unique Long COVID symptoms and/or experiences with treatments and medications Explore the treatment experiences that have been shared by others CURE ID, A Tool for Clinicians to Report New Uses of Existing Drugs with Heather Stone and Dr. Marco Schito Q&A with FDA Podcast | Transcript https://www.fda.gov/drugs/news-events-human-drugs/cure-id-tool-clinicians-report-new-uses-existing-drugs-heather-stone-and-dr-marco-schito?utm_medium=email&utm_source=govdelivery

Updated EULAR recommendations guide treatment decisions for rheumatoid arthritis

https://www.news-medical.net/news/20260313/Updated-EULAR-recommendations-guide-treatment-decisions-for-rheumatoid-arthritis.aspx First published in 2010, the EULAR recommendations for the management of RA, the most frequent inflammatory rheumatic disease, have been relied upon by healthcare professionals and organizations worldwide to offer an up-to-date and robust analysis of the effectiveness and practical use of available DMARDs – from conventional agents such as methotrexate to biologics and Janus kinase (JAK) inhibitors. The recommendations were last reviewed in 2022 to include key safety factors. The current fifth update was again based on reviews of the most recent evidence regarding these therapies and provides the most up-to-date guidance. Although there have been no new drugs approved since the last version, deepened insights as well as important strategic developments have accumulated.