miércoles, 21 de octubre de 2026
Microdosing LSD shows some cognitive benefits — but only for women Different effects of low-dose psychedelics on sustained attention in women and men deserve further investigation. By Gemma Conroy
https://www.nature.com/articles/d41586-026-03265-y?utm_source=Live+Audience&utm_campaign=b691f2a608-nature-briefing-daily-20261020&utm_medium=email&utm_term=0_-33f35e09ea-50432164
Chart showing the results of assessing the effects of the psychedelic drug LSD on cognitive function in a group of healthy participants: 24 women and 24 men. The drug significantly decreased the number of attention lapses that women experienced during a 10-minute task — an effect that wasn’t seen in men.
Tiny doses of the psychedelic drug LSD seem to boost some elements of cognition — but only in women. In a small trial, women who took 15 micrograms of the drug focused for longer periods and responded faster to a stimulus than did those who were given a placebo. This trial alone can’t confirm that LSD was behind these benefits, but the signal “warrants further investigation”, says psychopharmacologist Mikkel Højlund.
Will this sleep-disorder drug be the next big blockbuster? Orexin agonists have been approved for the treatment of a type of narcolepsy, yet they could also treat conditions across the health spectrum. By Amanda Heidt
https://www.nature.com/articles/d41586-026-03310-w?utm_source=Live+Audience&utm_campaign=b691f2a608-nature-briefing-daily-20261020&utm_medium=email&utm_term=0_-33f35e09ea-50432164
In August, a type of drug called an orexin agonist was approved by US regulators to treat narcolepsy — the first of what researchers hope will be many uses for this class of drug. Several pharma companies are already exploring the effectiveness of orexin agonists to treat conditions beyond sleep disorders, such as attention deficit hyperactivity disorder (ADHD). “Diabetes was the tip of the iceberg for GLP-1s, and I’m convinced the same story will play out for orexin,” says Andrew Plump, president of research and development at pharmaceutical company Takeda.
Longevity Boom: How to Respond When ‘Normal’ Isn’t Enough (+++ +++)
Longevity Boom: How to Respond When ‘Normal’ Isn’t Enough
Medscape Medical News
Outside My Specialty
https://www.medscape.com/viewarticle/longevity-boom-how-respond-when-normal-isnt-enough-2026a10012h5?ecd=wnl_oms_ous_all_en_etid8811805_261019_1&uac=148436CN&impID=8811805
How Are Stress, Depression, and Cardiovascular Risk Linked?
Medscape News Europe
Outside My Specialty
https://www.medscape.com/viewarticle/how-are-stress-depression-and-cardiovascular-risk-linked-2026a10012b3?ecd=wnl_oms_ous_all_en_etid8811805_261019_2&uac=148436CN&impID=8811805
Beyond CGRP: The Search for Migraine’s Next Big Target
Medscape Medical News
Outside My Specialty
https://www.medscape.com/viewarticle/beyond-cgrp-search-migraines-next-big-target-2026a100125z?ecd=wnl_oms_ous_all_en_etid8811805_261019_3&uac=148436CN&impID=8811805
Low-Calorie Diet/Exercise Intervention May Drive Diabetes Into Remission
Medscape Medical News
Outside My Specialty
https://www.medscape.com/viewarticle/low-calorie-diet-exercise-intervention-may-drive-diabetes-2026a10012cw?ecd=wnl_oms_ous_all_en_etid8811805_261019_4&uac=148436CN&impID=8811805
Medscape Poll: New Targets in Migraine: Where Do They Fit in Your Practice?
Medscape Reader Polls
Outside My Specialty
https://www.medscape.com/viewarticle/medscape-poll-new-targets-migraine-where-do-they-fit-your-2026a100120m?ecd=wnl_oms_ous_all_en_etid8811805_261019_5&uac=148436CN&impID=8811805
Functional Dyspepsia or GLP-1 Adverse Effect? How to Tell
Medscape Europe
https://www.medscape.com/viewarticle/functional-dyspepsia-or-glp-1-adverse-effect-how-tell-2026a10012c7?ecd=wnl_oms_ous_all_en_etid8811805_261019_6&uac=148436CN&impID=8811805
How knocking on doors can help beat measles — again America pushed back on diseases by building trust door by door. Let’s go back to that model
https://www.statnews.com/2026/10/21/measles-public-health-door-to-door-initiatives/
By Raj PanjabiOct. 21, 2026
Panjabi is a Harvard Medical School and Mass General Brigham physician, adviser to the Governors Public Health Alliance, and former White House senior director for global health security and biodefense at the National Security Council.
Replication is necessary even if scientists do everything right The NIH is pulling back on plans to prioritize replication. That’s a bad idea
https://www.statnews.com/2026/10/21/nih-replication-process-type-1-error-bhattacharya/
By Mark Louie F. RamosOct. 21, 2026
Ramos is an assistant research professor in the Department of Health Policy and Administration at Pennsylvania State University.
Patients Taking GLP-1s Face a Double Dose of Stigma
https://www.medscape.com/viewarticle/patients-taking-glp-1s-face-double-dose-stigma-2026a10012zi?ecd=WNL_trdalrt_pos1_ous_261020_etid8813672&uac=148436CN&impID=8813672
GLP-1 medications have surged in popularity, transforming treatment for obesity and diabetes and showing promise for a growing range of other conditions. According to Gallup, an estimated 11% of US adults are currently taking GLP-1s for weight loss, nearly four times the rate since 2024.
But despite their clinical benefits, people taking GLP-1s can face a double stigma: criticism related to their body size and judgment for using medication to lose weight. These attitudes may influence whether patients disclose their GLP-1 use, how they discuss treatment with others, and even their healthcare decisions.
FDA Approves Once-Weekly Generic Hormone Therapy Patch for Women (+++)
https://www.fda.gov/news-events/press-announcements/fda-approves-once-weekly-generic-hormone-therapy-patch-women?utm_medium=email&utm_source=govdelivery
The U.S. Food and Drug Administration yesterday approved the first generic of Climara Pro (estradiol and levonorgestrel transdermal system, 0.045 mg per day/0.015 mg), a transdermal hormone therapy patch for women with an intact uterus applied once a week. The approval aligns with FDA’s Public Health Pillar: Increasing Access to Affordable Medicines & Medical Products.
“Menopause is a universal experience for women, and it can greatly affect a person’s quality of life. This approval makes effective treatment more accessible,” said Acting FDA Commissioner Kyle Diamantas, J.D. “Approvals like this reflect FDA’s ongoing commitment to expanding American patient access to safe, affordable, high-quality medications.”
https://www.fda.gov/about-fda/what-we-do/fdas-public-health-pillars?utm_medium=email&utm_source=govdelivery
The generic estradiol and levonorgestrel transdermal system is therapeutically equivalent to Climara Pro, which is indicated for use in women with an intact uterus for:
Treatment of moderate to severe vasomotor symptoms due to menopause
Prevention of postmenopausal osteoporosis
Climara Pro and the generic estradiol and levonorgestrel transdermal system contain both estrogen and progestogen in a single patch; this differs from estrogen-only patches commonly used in menopause treatment. Climara Pro and the generic estradiol and levonorgestrel transdermal system are specifically approved for women with an intact uterus, as the progestogen component is included to reduce the risk of endometrial hyperplasia that can occur with estrogen use alone.
“Once-weekly dosing can make a real difference in how patients manage their care,” said Michael Davis, M.D., Ph.D., Director of FDA's Center for Drug Evaluation and Research. “A patch offers a convenient alternative to daily oral medications, which may help patients stay on track with their treatment.”
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/fda-drug-competition-action-plan?utm_medium=email&utm_source=govdelivery
Climara Pro and the generic estradiol and levonorgestrel transdermal system are contraindicated in patients with undiagnosed abnormal genital bleeding; breast cancer or a history of breast cancer; estrogen-dependent neoplasia; active deep vein thrombosis (DVT), pulmonary embolism (PE) or a history of these conditions; active arterial thromboembolic disease or a history of these conditions; known anaphylactic reaction or angioedema or hypersensitivity to estradiol and levonorgestrel; hepatic impairment or disease; and protein C, protein S, or antithrombin deficiency, or other thrombophilic disorders.
The most common adverse reactions (greater than or equal to 5%) are: application site reaction, vaginal bleeding, breast pain, upper respiratory infection, back pain, depression, pain, headache and flu syndrome.
In the U.S., nine out of 10 prescriptions filled are for generic drugs. Increasing the availability of generic drugs helps to create competition in the marketplace, which then helps to make treatment more affordable and increases access to healthcare for more patients. Learn more about the FDA Drug Competition Action Plan.
Climara Pro is a registered trademark of Bayer. The FDA approved Zydus Pharmaceuticals (USA) Inc.’s Estradiol and Levonorgestrel Transdermal System. Please contact the manufacturer for information about the medicine’s availability.
FDA Approves First-of-Its-Kind Chronic Hepatitis B Treatment New treatment may allow some patients to maintain viral control after stopping all hepatitis B therapy
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy?utm_medium=email&utm_source=govdelivery
The U.S. Food and Drug Administration today approved Hibsago (bepirovirsen) injection, in combination with existing antiviral therapy, to treat chronic hepatitis B virus (HBV) infection in certain adults without cirrhosis. Hibsago is the first FDA-approved antisense oligonucleotide to treat chronic hepatitis B. Hibsago is approved for certain adults without cirrhosis who have been taking HBV antiviral therapy for at least six months, have suppressed levels of HBV in their blood and have hepatitis B surface antigen (HBsAg) levels of 3,000 IU/mL or less, as determined by an FDA-authorized test.
“For many people living with chronic hepatitis B, treatment can mean taking antiviral medication for years to keep the virus suppressed,” said Wendy Carter, DO, Acting Director of the Office of Infectious Diseases in the Center for Drug Evaluation and Research. “Hibsago provides a new treatment approach that gives some patients the possibility of maintaining control of the virus after all chronic HBV treatment has ended. This approval represents an important advance for people living with this serious disease.”
Chronic hepatitis B is a long-term liver infection caused by HBV. Over time, the infection can lead to serious liver disease, including cirrhosis, and increase the risk of liver cancer. Hibsago works by targeting the genetic instructions the virus uses to make copies of itself and viral proteins, helping reduce the amount of virus and hepatitis B surface antigen in the body.
The FDA also authorized the Roche Elecsys HBsAg II quant II RxDx assay as a companion diagnostic device to aid in the selection of patients with chronic HBV infection for treatment with antiviral therapy in accordance with the FDA approved drug labeling and to assist in monitoring patients throughout the course of treatment.
Researchers studied Hibsago in two clinical trials that compared it with a placebo, an inactive treatment. Hibsago is injected under the skin for up to 24 weeks. Patients take it alongside their existing hepatitis B medicine and continue that medicine for at least 24 weeks after their last Hibsago injection. Some patients may then be able to stop their existing medicine if they meet certain health and lab test requirements.
Across the two trials, 24% of patients who received Hibsago met the requirements to stop their existing medicine, compared with none who received placebo. Twenty-four weeks after stopping all hepatitis B treatment, 19% of patients who received Hibsago still had the virus under control, and a protein that signals hepatitis B infection (HBsAg) could not be detected in their blood.
Hibsago has a boxed warning for serious liver reactions as well as low platelet counts, which can increase the risk of potentially life-threatening bleeding. Hibsago also has warnings for worsening kidney function, serious inflammatory adverse reactions like vasculitis, and serious risks if the injection is not administered properly. Patients need regular blood tests and follow up with their healthcare provider before and during treatment to monitor these risks. The most common side effects, excluding laboratory abnormalities, are injection site reactions, fever, fatigue and headache.
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track?utm_medium=email&utm_source=govdelivery
Optimizing Clinical Practice in Prostate Cancer (video series) +... +
Optimizing Clinical Practice in Prostate Cancer
Advanced prostate cancer (PC) includes metastatic hormone-sensitive PC (mHSPC), nonmetastatic castration-resistant PC (nmCRPC), and metastatic castration-resistant PC (mCRPC). Management of these disease types differs, but the overall goal when treating them is the same: to prolong life, to delay disease progression, and to maintain quality of life for patients by minimizing symptoms. This Learning Center will focus on understanding the latest data in advanced PC, including innovations in androgen receptor-targeted (ART) therapy, radiopharmaceuticals, how to interpret the data for clinical practice, and what impact this may have on clinical decision-making and aligning treatment for the individual patient.
https://www.medscape.org/advances/optimizing-clinical-practice-prostate-cancer-2026a1000jj9?src=mkmcmr_driv_32025_mscpedu_461680.01_ace_final&uac=148436CN
The New Era of mHSPC Treatment: Evaluating Emerging Doublet and Triplet Combinations
Authors: Neal D. Shore, MD, FACS; Sarah Caulfield, PharmD, BCOP; Rana R. McKay, MD, FASC
https://www.medscape.org/viewarticle/new-era-mhspc-treatment-evaluating-emerging-doublet-and-2026a1000kt7?page=1&src=mkmcmr_driv_32025_mscpedu_461680.01_ace_final&uac=148436CN
Medscape Now! Cancer and Bone Health Screening: Evidence-Based Strategies for Older Adults Authors: Blessing Ajayi, PA-C (++)
Medscape Now! Cancer and Bone Health Screening: Evidence-Based Strategies for Older Adults
Authors: Blessing Ajayi, PA-C
https://www.medscape.org/viewarticle/medscape-now-cancer-and-bone-health-screening-evidence-based-2026a10012vq?page=1&src=wnl_tpal_261020_mscpedu&uac=148436CN&impID=8815023
Understanding Cancer and Bone Health Challenges in Aging Populations
Population aging is a defining global trend, driven by longer lives, improved survival, and lower fertility.[1] The global population aged 65 years and older is projected to increase substantially, reaching about 17% of the world's population by 2050.[1] Cancer disproportionately affects older adults: More than 60% of patients with cancer are aged 65 years or older.[2] Knowledge gaps remain regarding the safest and most effective treatments for this population.[2] Aging increases bone loss in both women and men.[3] Estrogen deficiency is a major cause of accelerated bone loss; in breast cancer, estrogen deprivation decreases bone mineral density and raises fracture incidence by 40% to 50%.[3] Androgen-deprivation therapy (ADT) suppresses estrogen production and likewise accelerates bone loss and increases fracture rates.[3] Cytotoxic chemotherapy may also adversely affect bone metabolism.[3]
Medscape NOW! Prehabilitation Before Major Abdominal Surgery: Exercise, Nutrition, and Outcomes
Authors: Naseem Bazargan, MPH
https://www.medscape.org/viewarticle/medscape-now-prehabilitation-before-major-abdominal-surgery-2026a10012w8?page=1&src=wnl_tpal_261020_mscpedu&uac=148436CN&impID=8815023
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
Med Op-Ed: Time Toxicity, Clinical Trial Access, and RECIST Edited by Victoria Stern
https://www.medscape.com/viewarticle/med-op-ed-time-toxicity-clinical-trial-access-and-recist-2026a10013hh
In this installment of Med Op-Ed: The concept of time toxicity is morphing from patient- to stakeholder-centric, clinical trial access remains a challenge, and why it’s time to revisit Response Evaluation Criteria in Solid Tumors (RECIST).
Cutaneous T-Cell Lymphoma: Reduced US Incidence Rates Surprise Researchers John Jesitus (++) +...
Cutaneous T-Cell Lymphoma: Reduced US Incidence Rates Surprise Researchers
John Jesitus
https://www.medscape.com/viewarticle/cutaneous-t-cell-lymphoma-reduced-us-incidence-rates-2026a10013fc?src=
A recent analysis showed that although age-adjusted US rates of cutaneous T-cell lymphoma (CTCL) incidence rose between 2000 and 2015, the number of cases declined from 2015 to 2022 — a timeframe marked by widespread use of biologic drugs that have raised concerns about potentially inducing or exacerbating CTCL. As an epidemiologic study, the analysis did not assess causality. However, authors and a CTCL expert agreed that it is plausible that dupilumab in particular might have accounted for at least some of the downturn.
Watchful Waiting Has Lasting QOL Benefit in Rectal Cancer
https://www.medscape.com/viewarticle/watchful-waiting-has-lasting-qol-benefit-rectal-cancer-2026a10013eh?src=
Patients who are able to undergo watchful waiting instead of immediate surgery for locally advanced rectal cancer can see lasting gains in quality of life (QOL), according to a new analysis of two clinical trials.
https://www.medscape.com/index/list_13474_0
Blood-Based CRC Test Upgrades Improve on Precancerous Lesion Detection Nancy A. Melville
https://www.medscape.com/viewarticle/blood-based-crc-test-upgrades-improve-precancerous-lesion-2026a10013i5?src=
NASHVILLE, Tenn. — Updates to an FDA-approved colorectal cancer (CRC) blood test increased the detection of advanced precancerous lesions (APLs), a new study showed.
“The updated test has improved performance, better detection of advanced precancerous lesions, resulting in larger projected reductions in CRC incidence and mortality at the population level,” said first author Aasma Shaukat, MD, MPH, of the NYU Grossman School of Medicine in New York City.
EMA Declines Pediatric Extension for AML Drug Edited by Joyani Das (+++)
https://www.medscape.com/viewarticle/ema-declines-pediatric-extension-aml-drug-2026a10013lb?src=
Children with newly diagnosed acute myeloid leukemia (AML) in Europe will not gain a new targeted option for now. The European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has finalized its assessment of an application to extend the use of Mylotarg (gemtuzumab ozogamicin, Pfizer) to children aged 1 year to less than 18 years with newly diagnosed AML, in combination with cytarabine and mitoxantrone. The committee did not recommend the new use.
EMA Upholds Rejection of Posttransplant Complication Drug
Edited by Joyani Das
https://www.medscape.com/viewarticle/ema-upholds-rejection-posttransplant-complication-drug-2026a10013l6?src=
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has confirmed its recommendation to refuse marketing authorization for narsoplimab (Yartemlea, Omeros Ireland Limited) for hematopoietic stem cell transplant (HSCT)-associated thrombotic microangiopathy (TA-TMA). The opinion, adopted on October 15, 2026, follows a re-examination requested by the company after the committee’s initial negative opinion on June 25, 2026.
EMA Backs Oral Suspension for Malignant Brain Tumors
https://www.medscape.com/viewarticle/ema-backs-oral-suspension-malignant-brain-tumors-2026a10013l2?src=
At its October 2026 meeting, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has recommended Kizfizo (temozolomide, Orphelia Pharma) 40 mg/mL oral suspension for adults with newly diagnosed glioblastoma and for adults and children aged 1 year or older with recurrent or progressive malignant glioma.
Kizfizo is a hybrid medicine of Temodal, the capsule formulation authorized in the EU since January 26, 1999. It contains the same active substance. The new product differs in being a 40 mg/mL suspension, and it extends approval to patients aged 1 year to less than 3 years.
Oncology Cases & Quizzes
Woman With Breast Angiosarcoma Responds to Paclitaxel
An individualized paclitaxel strategy produced a complete response in one frail older woman with radiation-associated breast angiosarcoma.
Medscape Global, October 19, 2026
https://www.medscape.com/index/list_12039_0
FDA Approves Dostarlimab for Rectal Cancer
https://www.medscape.com/viewarticle/fda-approves-dostarlimab-rectal-cancer-2026a10013c7?ecd=WNL_trdalrt_pos1_ous_261021_etid8816935&uac=148436CN&impID=8816935
The FDA has granted accelerated approval to dostarlimab (Jemperli, GSK) for rectal cancer indications.
The drug is now approved for adults with stage II/III locally advanced rectal cancer (LARC) that is microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR).
The PD-1 inhibitor is the first immunotherapy to be approved specifically for MSI-H or dMMR rectal cancer, which accounts for 5%-10% of tumors. Based on the results of the approval study, AZUR-1, GSK said dostarlimab could help eligible patients avoid chemotherapy, radiation, and surgery.
I was told it was anxiety–but it was Wilson’s disease
https://rarerevolutionmagazine.com/i-was-told-it-was-anxiety-but-it-was-wilsons-disease/
Aneesha Premkumar’s journey to diagnosis spanned several years. Her symptoms were attributed to anxiety and stress, but it turned out to be Wilson’s disease. She describes her life before and after diagnosis and highlights the importance of advocating for yourself
Written by Aneesha Premkumar
When I was diagnosed with Wilson’s disease on 2 February 2024, my life finally started to make sense.
For a long time, I knew something wasn’t right, but I couldn’t understand why my body seemed to be changing in ways I couldn’t control. What started as small symptoms gradually became impossible to ignore.
My speech began to change. I found myself mumbling words and struggling to communicate clearly. My handwriting became smaller and harder to read after only a few sentences. I developed tremors, experienced brain fog, and found it difficult to remember things. Walking became harder, and there were days when I needed support just to get around.
martes, 20 de octubre de 2026
The Lancet Commission on 21st century global threats to health Prof Christopher J L Murray, DPhila,b,* Send email to cjlm@uw.edu ∙ Natalia Kanem, MDc,* ∙ Shameran Abed, Bar-at-Lawd ∙ Prof Ibrahim Abubakar, PhDe ∙ Ifedayo M Adetifa, PhDf,g,h ∙ Ximena P Aguilera, MDi ∙ et al.
The Lancet Commission on 21st century global threats to health
Prof Christopher J L Murray, DPhila,b,* Send email to cjlm@uw.edu ∙ Natalia Kanem, MDc,* ∙ Shameran Abed, Bar-at-Lawd ∙ Prof Ibrahim Abubakar, PhDe ∙ Ifedayo M Adetifa, PhDf,g,h ∙ Ximena P Aguilera, MDi ∙ et al.
https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01714-9/abstract?utm_campaign=conferencealerts&utm_medium=email&dgcid=hubspot_email_conferencealerts_whs26&_hsenc=p2ANqtz-8E2GSnz-zYNMa5ENjEvwqERZNe7ggCA8Mp-tDGeubMB0pJGBadraZGwnxkcCeU5ffZrBDOf_iExFcHqQI4jcQ9J-5n2w&_hsmi=443908007&utm_source=hs_email
Editorial
p1525
Rethinking threats to human survival
The Lancet
https://www.thelancet.com/journals/lancet/issue/vol408no10564/PIIS0140-6736(26)X2039-6
Cancer-related depression among Southwest American Indian survivors: a qualitative study Rey Paolo Ernesto J. Roca III1 , Wendie Robbins1 , Felicia Schanche Hodge1,2, (+ +...)
Cancer-related depression among Southwest American Indian survivors: a qualitative study
Rey Paolo Ernesto J. Roca III1, Wendie Robbins1, Felicia Schanche Hodge1,2,
https://www.academia.edu/3071-0324/2/3/10.20935/AcadPHealth8402?email_action=download
Introduction: Depression is common among cancer survivors and may disrupt sleep, appetite, and daily functioning. American Indian and Alaska Native populations experience disproportionate cancer incidence and mortality for several cancers, including cervical, liver, and stomach cancer. These disparities are shaped by well-documented structural barriers, including limited access to quality care and a shortage of culturally responsive mental health services in American Indian and Alaska Native (AI/AN) communities. Limited research has examined how Indigenous cancer survivors understand and respond to depression within the context of cancer survivorship.
https://www.academia.edu/3071-0324/2/3/10.20935/AcadPHealth8402
Long sleep duration and mortality among U.S. cancer survivors: NHIS-NDI linkage
Tolulope Adebile, Purbasha Biswas, Temitayo Paul, Olamide Asifat, Xinyan Zhang, Jian Zhang, Lili Yu
Volume 2, Issue 3
https://www.academia.edu/journals/academia-global-and-public-health/articles?source=journal-top-nav
Making dementia clinical trials more representative The Lancet Healthy Longevity (+...)
Making dementia clinical trials more representative
The Lancet Healthy Longevity
https://www.thelancet.com/journals/lanhl/issue/vol7no9/PIIS2666-7568(26)X2009-8
CAR T cells in autoimmunity: safety first The Lancet Rheumatology (+...)
CAR T cells in autoimmunity: safety first
The Lancet Rheumatology
https://www.thelancet.com/journals/lanrhe/issue/vol8no11/PIIS2665-9913(26)X2010-0
Viewpoint: Why taxes, regulations, and scare tactics won’t convert people into eating healthier Economist | October 20, 2026
https://geneticliteracyproject.org/2026/10/20/viewpoint-why-taxes-regulations-and-scare-tactics-wont-convert-people-into-eating-healthier/
Decades of gentle nudging haven’t slowed the global obesity epidemic, so governments are getting pushier — slapping warning labels on junk food, taxing it, and restricting where it can be sold, as The Economist reports.
Vaccines, religious freedom, and protecting children’s health: New York Amish appeal to Supreme Court after $118,000 in vaccine fines for blocking shots Reason | October 20, 2026
https://geneticliteracyproject.org/2026/10/20/vaccines-religious-freedom-and-protecting-childrens-health-new-york-amish-appeal-to-supreme-court-after-118000-in-vaccine-fines-for-blocking-shots/
New York’s Amish are taking their fight over school vaccine mandates to the US Supreme Court, after the state hit three rural Amish schoolhouses with $118,000 in fines for enrolling unvaccinated students, as Reason’s Ryan Curtis reports.
Challenging creation of a ‘vaccine injury clinic,’ NIH research scientists rebuke Kennedy for politicizing and undermining science New York Times | October 20, 2026
https://geneticliteracyproject.org/2026/10/20/challenging-creation-of-a-vaccine-injury-clinic-nih-research-scientists-rebuke-kennedy-for-politicizing-and-undermining-science/
The board that oversees the National Institutes of Health’s research hospital has publicly rebuked Health Secretary Robert F. Kennedy Jr., voting unanimously to back a statement from its chairman accusing Kennedy of undermining trust in science and politicizing the hospital’s work, as The New York Times’ Sheryl Gay Stolberg reports.
Testosterone’s growing appeal to women: It anecdotally mildly improves sex life, but are there any other potential benefits—or red flags? Daniel Kelly | October 20, 2026
https://geneticliteracyproject.org/2026/10/20/testosterones-growing-appeal-to-women-it-anecdotally-mildly-improves-sex-life-but-are-there-any-other-potential-benefits-or-red-flags/
A recent BBC report described women posing as men to get hold of testosterone gel, hoping to improve their menopause symptoms, because female testosterone products are not easily available to them. But why is the appetite for this predominantly male hormone exploding and are there risks involved?
RFK Jr.’s EPA capitulates to anti-abortionists, authorizes study of safe abortion pills which activists falsely claim are “poisoning” our water supply Jenae Barnes | October 20, 2026
https://geneticliteracyproject.org/2026/10/20/rfk-jr-s-epa-capitulates-to-anti-abortion-activists-authorizes-study-of-safe-abortion-pills-mifepristone-and-misoprostol-which-they-claim-are-poisoning-americas-w/
After pressure from anti-abortion advocates, Republican lawmakers and state officials, the Environmental Protection Agency announced this month that it will launch a study to test for trace amounts of abortion pills as a water contaminant in multiple cities. Scientists say there’s no evidence that mifepristone and misoprostol, the two-drug regimen that is highly effective in ending a pregnancy, harms the environment or people.
FDA Approves Gazyva (Obinutuzumab) for Treatment of Childhood-Onset Idiopathic Nephrotic Syndrome by Madaline Spencer| Published on: Oct 1, 2026
https://checkrare.com/fda-approves-gazyva-obinutuzumab-for-treatment-of-childhood-onset-idiopathic-nephrotic-syndrome/
The US Food and Drug Administration (FDA) has approved Gazyva (obinutuzumab) to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or steroid dependent childhood-onset idiopathic nephrotic syndrome (INS) who are in complete remission.
INS is a rare, autoimmune kidney disease characterized by unpredictable relapses that cause fatigue, swelling, weight gain and increased susceptibility to infections and clotting, as well as anxiety, depression and reduced self-esteem, brought on by the fear of relapse and social isolation. It is usually diagnosed in early childhood and is the most common form of primary glomerular disease in children.
FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option
by Madaline Spencer| Published on: Oct 14, 2026
Keisha L. Gibson, MD, MPH, pediatric nephrologist at the UNC School of Medicine, discusses the approval of Gazyva (obinutuzumab) for patients with childhood-onset idiopathic nephrotic syndrome (INS).
https://checkrare.com/fda-approves-gazyva-for-idiopathic-nephrotic-syndrome-offering-a-new-treatment-option/
FDA Approves Jaypirca (Pirtobrutinib) for Two Rare Blood Cancers by Madaline Spencer| Published on: Oct 13, 2026
https://checkrare.com/fda-approves-jaypirca-pirtobrutinib-for-two-rare-blood-cancers/
The US Food and Drug Administration (FDA) has approved pirtobrutinib for adults with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) with no known 17p deletion.
CLL/SLL are rare cancers that affect B lymphocytes. The conditions are similar in pathophysiology but differ primarily in location of the disease. In patients with CLL, the cancer mainly manifests in the bloodstream, while in patients with SLL, it is in the lymph nodes and lymphoid tissue. CLL accounts for the majority (90%) of cases. Early signs and symptoms may include swollen lymph nodes, fatigue, weight loss, fever, night sweats and/or frequent...
KAT6 Syndromes by CheckRare Staff| Published on: Sep 24, 2026 KAT6 syndromes are rare genetic neurodevelopmental disorders caused by genetic mutations in the KAT6A or KAT6B genes.
KAT6 Syndromes
by CheckRare Staff| Published on: Sep 24, 2026
KAT6 syndromes are rare genetic neurodevelopmental disorders caused by genetic mutations in the KAT6A or KAT6B genes.
https://checkrare.com/kat6-syndromes/
Breakthrough Therapy Designation to Cimzia for Antiphospholipid Syndrome
FDA Grants Breakthrough Therapy Designation to Cimzia for Pregnancy Complications in Antiphospholipid Syndrome
by Madaline Spencer| Published on: Oct 16, 2026
https://checkrare.com/fda-grants-breakthrough-therapy-designation-to-cimzia-for-pregnancy-complications-in-antiphospholipid-syndrome/
Jane Salmon, MD, Professor of Medicine at Weill Cornell Medical College, discusses antiphospholipid syndrome (APS), its impact on pregnancy, and emerging research into Cimzia (certolizumab pegol) as a potential approach to preventing serious pregnancy complications.
Efficacy of Infigratinib on Achondroplasia by Madaline Spencer| Published on: Oct 19, 2026
Efficacy of Infigratinib on Achondroplasia
by Madaline Spencer| Published on: Oct 19, 2026
Daniela Rogoff, MD, PhD, Chief Medical Officer, Skeletal Dysplasia of BridgeBio, discusses the efficacy of infigratinib to treat persons with achondroplasia.
https://checkrare.com/efficacy-of-infigratinib-on-achondroplasia/
Efficacy of Vosoritide to Treat Hypochondroplasia by Madaline Spencer| Published on: Oct 20, 2026
Efficacy of Vosoritide to Treat Hypochondroplasia
by Madaline Spencer| Published on: Oct 20, 2026
Andrew Dauber, MD, MMsc, Chief of Endocrinology at Children’s National, discusses the efficacy of vosoritide to treat persons with hypochondroplasia.
https://checkrare.com/efficacy-of-vosoritide-to-treat-hypochondroplasia/
Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11 by CheckRare Staff| Published on: Oct 14, 2026
Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11
by CheckRare Staff| Published on: Oct 14, 2026
In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct clinical trials.
https://checkrare.com/ultragenyx-sells-prv-for-210m-the-checkrare-brief-ep-11/
Classical homocystinuria (HCU) HCU—2027 and beyond!
https://rarerevolutionmagazine.com/digitalspotlight/hcu-2027-and-beyond/
Written by Karen Roberts and Nicola Miller, RARE Revolution
Interview with
Tara Morrison, director of HCU Network Australia,
Danaé Bartke, executive director HCU Network America
Sarah Jones, community lead, Metabolic Support
Classical HCU causes a deficiency of cystathionine beta-synthase (CBS). Without enough CBS, the amino acids homocysteine and methionine build up in the blood. HCU can cause complications such as stroke, pulmonary embolism and deep vein thrombosis, ophthalmologic and skeletal complications, as well as developmental delay. Treatment may consist of vitamin supplementation, dietary restrictions, medication and an amino acid formula. Newborn screening for the condition is available, albeit inconsistent globally, and cases of missed diagnoses have been reported.
An RNA splicing factor could help reverse cellular aging A machine learning-based screen identified SRSF1 as a potential target for reversing several hallmarks of aging without erasing cell identity, with further testing revealing benefits in aged mice. Written byBree Foster, PhD
An RNA splicing factor could help reverse cellular aging
A machine learning-based screen identified SRSF1 as a potential target for reversing several hallmarks of aging without erasing cell identity, with further testing revealing benefits in aged mice.
Written byBree Foster, PhD
https://www.drugdiscoverynews.com/an-rna-splicing-factor-could-help-reverse-cellular-aging-17593
Aging gradually erodes the function of cells, tissues, and organs, increasing the risk of chronic diseases such as cardiovascular disease, neurodegeneration, and cancer. Researchers are therefore investigating ways to reverse age-related changes without disrupting normal cellular function.
FDA grants accelerated approval to dostarlimab for locally advanced rectal cancer
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-dostarlimab-locally-advanced-rectal-cancer?utm_medium=email&utm_source=govdelivery
On October 19, 2026, the Food and Drug Administration granted accelerated approval to dostarlimab (Jemperli, GlaxoSmithKline LLC) for adults with Stage II/III locally advanced rectal cancer (LARC) that is microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR), as determined by an FDA authorized test for solid tumors.
lunes, 19 de octubre de 2026
Digital Breast Tomosynthesis Screening Reduces Interval Cancers by 24% but Not Significantly
https://www.medscape.com/viewarticle/digital-breast-tomosynthesis-screening-reduces-interval-2026a10012r5?src=
Digital breast tomosynthesis (DBT) plus synthesised mammography lowered the 24-month invasive interval cancer rate by 24% compared with digital mammography among women aged 50-70 years in a randomised screening trial, but the difference was not statistically significant.
Intratumoral Immunotherapy Draws Renewed Attention but Does It Work? Kate Johnson
https://www.medscape.com/viewarticle/intratumoral-immunotherapy-draws-renewed-attention-does-it-2026a10012vx
After years on the margins, intratumoral immunotherapy is drawing renewed attention following the FDA’s recent approval of vusolimogene oderparepvec (Tudriqev, Replimune), the second immunotherapy approved for intratumoral use.
The drug is approved in combination with nivolumab to treat advanced melanoma that has progressed after an anti-PD-1-containing regimen.
Yoghurt, coffee and chocolate have something in common, and researchers mapped it across Europe
https://www.news-medical.net/news/20261018/Yoghurt-coffee-and-chocolate-have-something-in-common-and-researchers-mapped-it-across-Europe.aspx
From yoghurt and sourdough to coffee and chocolate, a survey spanning 53 European countries examines which fermented foods people consume and how much they actually eat.
Could ultraprocessed foods affect your brain before you notice?
https://www.news-medical.net/news/20261019/Could-ultraprocessed-foods-affect-your-brain-before-you-notice.aspx
Could what's on your plate affect your brain before your memory shows any signs of trouble? A study of nearly 9,000 Canadian adults links higher ultraprocessed food intake to greater body fat and signs of silent vascular brain injury, although the brain findings warrant caution.
Menopause isn't the end of the story for the ovaries
https://www.news-medical.net/news/20261019/Menopause-isnt-the-end-of-the-story-for-the-ovaries.aspx
Long after their reproductive role ends, ovaries continue to undergo molecular changes linked to inflammation, tissue remodeling, and cellular signaling, raising new questions about their potential influence on women's health as they age.
How GLP-1 Drugs May Help Treat Addiction: Mechanisms and Clinical Evidence
https://www.news-medical.net/health/How-GLP-1-Drugs-May-Help-Treat-Addiction-Mechanisms-and-Clinical-Evidence.aspx
Shared pathways linking metabolism, reward, and substance seeking are prompting scientists to investigate diabetes and obesity drugs as potential addiction treatments.
Substance use disorders (SUDs) represent a substantial global health burden, with an estimated 400 million people living with alcohol use disorder (AUD) worldwide reported in 2019.2 The high relapse rates of SUDs and limited pharmacological options for some substances support the need for additional treatments, alongside wider access to established care.
GLP-1 Drugs and Obstructive Sleep Apnea: How Weight Loss Medications Improve Breathing During Sleep
https://www.news-medical.net/health/GLP-1-Drugs-and-Obstructive-Sleep-Apnea-How-Weight-Loss-Medications-Improve-Breathing-During-Sleep.aspx
What can treatments targeting weight and metabolism offer people with obstructive sleep apnea, and which questions must clinical trials still answer?
Obstructive sleep apnea (OSA) is characterized by recurrent collapse of the upper airway during sleep, causing repeated airflow obstruction and leading to loud snoring, frequent awakenings, and excessive daytime sleepiness. Over time, fragmented sleep and recurrent nocturnal awakenings contribute to chronic fatigue, reduced energy levels, and impaired quality of life.
Gut Microbiome and Aging: How Gut Bacteria Influence Health and Longevity
Gut Microbiome and Aging: How Gut Bacteria Influence Health and Longevity
https://www.news-medical.net/health/Gut-Microbiome-and-Aging-How-Gut-Bacteria-Influence-Health-and-Longevity.aspx
From microbial shifts in later life to the distinctive gut communities of centenarians, researchers are investigating what the intestinal microbes reveal about how people age.
The gut microbiota comprises a diverse community of microorganisms that regulate metabolism, support immune function, preserve intestinal barrier integrity, and influence inflammatory responses throughout life. Disruptions in gut microbiome composition can alter microbial homeostasis, the balance of microbial activity within the gut, increase inflammation, and affect the risk of age-related diseases.2
Could this flowering plant help people lose body fat?
https://www.news-medical.net/news/20261019/Could-this-flowering-plant-help-people-lose-body-fat.aspx
A 12-week clinical trial found that adults taking Lythrum salicaria extract lost body fat while placebo recipients gained it, offering early evidence of a potential plant-based approach to weight management.
A recent Nutrients study evaluated the efficacy and safety of Lythrum salicaria extract supplementation in reducing body fat in adults with a body mass index (BMI) of 23–30 kg/m².
GLP-1s and Skin Cancer: What Dermatologists Should Know (+...)
https://www.medscape.com/c32/p14/cutis-october-2026-2026a10010ly?ecd=mkm_ret_261017_mscpmrk_derm_cutis-top-content_etid8804153&uac=148436CN&impID=8804153
The Role of Antidiabetic Drugs in Cutaneous Malignancies: An Evidence-Based Review
Seanna Yang, MD; Nickoulet Babaei, MD; Dahyeon Kim, MD; Mireya Cervantes, MD; Minka Gill, MD; Jashin J. Wu, MD
https://www.medscape.com/viewarticle/role-antidiabetic-drugs-cutaneous-malignancies-evidence-2026a100106u?ecd=mkm_ret_261017_mscpmrk_derm_cutis-top-content_etid8804153&uac=148436CN&impID=8804153
Caribou halts its allogeneic CAR T programs as funding dries up Facing a tight financing market, the CRISPR biotech will wind down two off-the-shelf cell therapies as investor money shifts toward in vivo CAR T. Written byAndrea Corona
Caribou halts its allogeneic CAR T programs as funding dries up
Facing a tight financing market, the CRISPR biotech will wind down two off-the-shelf cell therapies as investor money shifts toward in vivo CAR T.
Written byAndrea Corona
https://www.drugdiscoverynews.com/caribou-halts-its-allogeneic-car-t-programs-as-funding-dries-up-17584?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-_VKkkojNK1QpvONG19i16LDx1hyhVxpBuGF9WqsjO_WAAxM-6O8sg2-UCZz0I2nOOLWiRPOzAFFRmK9cTfxw-i_CrfSg&_hsmi=444630187&utm_content=444630187&utm_source=hs_email
For years, allogeneic CAR T cell therapy carried one of the field’s biggest promises: cancer-fighting cells made from healthy donors, stocked in advance, and ready the moment a patient needed them. Caribou Biosciences built its pipeline around that vision, using CRISPR genome editing to engineer donor cells that could persist and fight tumors. On October 6th, the company announced it would shelve both of its allogeneic chimeric antigen receptor (CAR) T cell therapy programs, including one ready for a Phase 3 trial.
Enhancing Clinical Understanding of Oral SERD-Based Therapies for Patients With ER-Positive/HER2-Negative MBC Authors: Nadia Harbeck, MD, PhD; Virginia Kaklamani, MD, DSc; Gregory Vidal, MD, PhD
Enhancing Clinical Understanding of Oral SERD-Based Therapies for Patients With ER-Positive/HER2-Negative MBC
Authors: Nadia Harbeck, MD, PhD; Virginia Kaklamani, MD, DSc; Gregory Vidal, MD, PhD
https://www.medscape.org/viewarticle/enhancing-clinical-understanding-oral-serd-based-therapies-2026a1000a46?page=1&src=mkmcmr_driv_stan_mscpedu_261018-OUS-HONC-enhancing-clinical-understanding-oral-serd-based-therapies-2026a1000a46-cta&uac=148436CN
Menopause (+++)...
https://womenshealth.gov/menopause?utm_medium=email&utm_source=govdelivery
Menopause is a normal stage of life. Symptoms are real, and treatments can help. Find current information here to help you when talking with your health care provider.
Today is the first day of National Women’s Blood Pressure Awareness Week (NWBPAW)! From October 18 to 24, the U.S. Department of Health and Human Services, Office on Women’s Health highlights priorities for supporting women’s heart health and encourages women to take charge of their blood pressure.
The 2026 theme, “Strong Hearts: Everyday Actions for Blood Pressure Control and Lifelong Health,” emphasizes making heart‑healthy habits part of everyday life. The theme highlights practical actions women can take to manage healthy blood pressure and protect their heart health while reinforcing the connection between blood pressure and chronic disease risk across the lifespan. It also highlights key life transitions, including pregnancy, the postpartum period, and perimenopause/menopause, when blood pressure can change.
https://www.cdc.gov/pregnancy/after/index.html?utm_medium=email&utm_source=govdelivery
This year’s focus areas include:
Nutrition & Blood Pressure: Putting Healthy Eating into Practice
Lifestyle & Digital Wellness: Everyday Behaviors that Influence Heart Health
Prevention & Women’s Heart Health: Reducing Risk Across the Life Course
Reversing Chronic Conditions: Improving Health Through Prevention & Action
National Women’s Blood Pressure Awareness Week 2026
https://womenshealth.gov/nwbpaw?utm_medium=email&utm_source=govdelivery
CURATIVE POTENTIAL NOW IN 2L DLBCL
https://global.columvi-hcp.com/?cid=na1191708001&utm_source=medscape&utm_medium=na&utm_campaign=columvi2026&utm_content=tx%3Aagenative%3A2603%3Axx&dclid=CjkKEQjw_9bWBhDiscOaku6CnKkBEiQAKCg80nE2km_tXx8jxv0bSnvbxCJY14soZkwxNvGTx15fUMTw_wcB&gad_source=7&gad_campaignid=23680757487
At 3 years, regardless of your patient’s age or relapse time in 2L DLBCL, COLUMVI + GemOx demonstrates sustained survival benefit. And it’s well tolerated. Be confident with the first and only approved off-the-shelf therapy with curative potential in 2L DLBCL
https://www.medscape.com/invitation/viewTracker.do?src=inve&uac=148436CN
PHESGO: Proven efficacy and a convenient method of administration Subcutaneous administration in minutes, without compromising on efficacy
HER2-targeted therapies have transformed the HER2+ breast cancer landscape
Give your patients a proven treatment approach that balances efficacy with well-established safety profiles, keeping your patients' quality of life at the centre of your treatment approach.
https://global.phesgo-hcp.com/?cid=na7759503509&utm_source=medscape&utm_medium=na&utm_campaign=her2_2026&utm_content=tx%3Aphesgonc5%3A2609%3Axx&dclid=CjkKEQjw_9bWBhDiscOaku6CnKkBEiQAKCg80ktc7Kj_Slf6KPtBQnPnT8UVfo34wD9aElkpGuCBeofw_wcB&gad_source=7
Reshaping Gastroesophageal Adenocarcinoma Care: Integrating New Anti-HER2 Therapies CME/CNE/CPE/IPCE Monday, November 9, 2026 | 3:30 pm - 4:15 pm ET Virtual
https://na.eventscloud.com/website/99132/
Transform your gastroesophageal adenocarcinoma (GEA) practice by joining expert oncologists Elena Elimova, Samuel J. Klempner, and Kohei Shitara for an interactive, 45-minute online discussion. Unpack the latest clinical trial data for novel anti-human epidermal growth factor receptor 2 (HER2) regimens, learn how to integrate new frontline regimens into care for patients with HER2-positive GEA, and gain insight into postprogression strategies.
Faculty will also discuss proactive team-based approaches to toxicity management. Register now to elevate your clinical decision-making.
The Fifth Transatlantic Exchange in Oncology: Transatlantic Innovations in Cancer Drug Research – A Selection of Key Presentations From the Event (video series)
https://www.medscape.com/medicalaffairs/448002.4/media-5?src=0_nl_sm_0&uac=148436CN
The Fifth Annual Transatlantic Exchange in Oncology reflects an ongoing collaboration between key scientists, clinicians, and researchers from the Institut Gustave Roussy in Villejuif, France and Dana-Farber Cancer Institute (DFCI) in Boston, USA (supported by L’Institut Servier). This year’s event brought together a global audience with expertise in medical oncology, translational research, and molecular biology to exchange and discuss insights and new developments in the rapidly evolving area of cancer drug research. The event was held in Boston – at the Jimmy Fund Auditorium at DFCI - on March 27, 2026. Prof Eric Fischer from DFCI gave his insights on the day with this short “Expert Commentary” on the program. Prof Toni Choueiri then provides the Welcome Address. This is followed by 4 videos from the day’s events which reflect the depth and diversity of cancer drug research and collaboration across the Atlantic.
The Rock's protein-heavy post-workout smoothie may not be right for everyone
The Rock's protein-heavy post-workout smoothie may not be right for everyone
https://www.foxnews.com/lifestyle
The Rock's protein-heavy post-workout smoothie may not be right for everyone
Dietitian says star's protein-packed drink offers nutritional benefits, but some ingredients may be excessive for others
By Sadie Chafe Fox News
Published October 19, 2026 6:00am EDT | Updated October 19, 2026 6:30am EDT
https://www.foxnews.com/food-drink/rocks-protein-heavy-post-workout-smoothie-may-not-right-everyone
Popular foods Americans eat every day linked to silent brain damage, alarming study finds
Popular foods Americans eat every day linked to silent brain damage, alarming study finds
https://www.foxnews.com/health
Researchers say the link between ultra-processed foods and brain health problems may be detectable before symptoms appear
By Sadie Chafe Fox News
Published October 18, 2026 1:07pm EDT | Updated October 18, 2026 1:39pm EDT
https://www.foxnews.com/health/popular-foods-americans-eat-every-day-linked-silent-brain-damage-alarming-study-finds
domingo, 18 de octubre de 2026
MAHA moms divided over RFK Jr.’s food‑safety efforts, as recall alerts grow and outbreaks spread USA Today | October 15, 2026
https://geneticliteracyproject.org/2026/10/15/maha-moms-divided-over-rfk-jr-s-food-safety-efforts-as-recall-alerts-grow-and-outbreaks-spread/
The “crunchy moms” who helped power Robert F. Kennedy Jr.’s Make America Healthy Again movement are splintering, as a year of food recalls and the nation’s largest recorded Cyclospora outbreak push some to turn on the Health and Human Services secretary, as Alyssa Goldberg reports for USA Today.
Retreat from identity labels: Does the sharply declining number of young teens identifying as gay and bisexual reflect an ideological shift? Washington Post | October 15, 2026
https://geneticliteracyproject.org/2026/10/15/retreat-from-identity-labels-does-the-sharply-declining-number-of-young-teens-identifying-as-gay-and-bisexual-reflect-an-ideological-shift/
The share of young teenagers identifying as lesbian, gay or bisexual has dropped sharply in just four years — and nobody is quite sure why. As the Washington Post’s Casey Parks reports, a new analysis of Centers for Disease Control and Prevention data finds the steepest decline among ninth-grade girls: 30 percent identified as lesbian or bisexual in 2021, compared with 17 percent in 2025.
Anti-vaxxers’ double whammy: Exaggerated vaccine fears have fueled measles outbreaks and preventable deaths—and left survivors more vulnerable to other infections for years Liz Szabo | October 15, 2026
https://geneticliteracyproject.org/2026/10/15/anti-vaxxers-double-whammy-exaggerated-vaccine-fears-have-fueled-measles-outbreaks-and-preventable-deaths-and-left-survivors-more-vulnerable-to-other-infections-for-years/
Measles infections make people miserable for up to two long weeks, commonly causing dangerously high fevers, a painful sensitivity to light and, of course, a tell-tale red rash.
Sufferers who escape without developing pneumonia, brain swelling, or other serious complications may believe the worst is over.
Viewpoint: Wellness patches: Most vitamin supplements are useless and sometimes even dangerous, but this craze is even worse Katie Suleta | October 15, 2026
https://geneticliteracyproject.org/2026/10/15/viewpoint-wellness-patches-most-vitamin-supplements-are-useless-and-sometimes-even-dangerous-but-this-craze-is-even-worse/
Promoted as high-tech nutrient delivery, over-the-counter vitamin patches are rapidly gaining popularity among consumers looking to bypass daily capsules. However, these products straddle a major regulatory loophole. When coupled with the impenetrable nature of the skin barrier, the science reveals these patches deliver far more marketing hype than active vitamins.
Vaccine-preventable congenital infections are a leading driver of autism Sarah Boden | October 16, 2026
https://geneticliteracyproject.org/2026/10/16/vaccine-preventable-congenital-infections-are-a-leading-driver-of-autism/
Kids born with certain congenital infections are approximately three times more likely to be diagnosed as having autism, according to a study out of the Karolinska Institutet in Stockholm, Sweden. These children were also more likely to have an intellectual disability compared to those born without an infection, with the risk of severe to profound intellectual disability being up to 30 times higher than the general population.
Kickstart Your Advocacy Webinar #3: Practical Advocacy: demystifying European institutions : #3 – Practical Advocacy: demystifying European institutions 🗓️ Date: 26 November 2026, 4 PM CEST 📍 Where: Online
https://www.eurordis.org/webinar-series-kickstart-your-advocacy/
Webinar Series: Kickstart your advocacy
August 2026
Young people have an important role to play in shaping the future for the rare disease community. But finding your voice, navigating traditional advocacy spaces, and knowing how to turn your ideas into action isn’t straightforward.
PLUTO PROJECT – Disregarded Rare Diseases IRDiRC - PLUTO PROJECT – Disregarded Rare Diseases
https://irdirc.org/pluto-project-disregarded-rare-diseases-2/
Unveiling the depth of the knowledge gap in the Universe of Rare Diseases: the PLUTO mission
This article presents findings from the PLUTO mission, which explores disparities in research knowledge and drug development across rare diseases. The authors, including Virginie Hivert, our Head of Therapies and Access, collaborated within the International Rare Diseases Research Consortium (IRDiRC) to examine the underrepresentation of non-oncological rare diseases in research and development. The article also proposes a new way of visualising the ‘Rare Disease Universe’.
https://link.springer.com/article/10.1186/s13023-026-04574-1
Engaging children, adolescents and young adults living with rare diseases as research partners: practical considerations for meaningful involvement
https://www.frontiersin.org/journals/pediatrics/articles/10.3389/fped.2026.1886270/full
Engaging children, adolescents and young adults living with rare diseases as research partners
This recent IRDiRC article explores the importance of engaging young people living with rare diseases as research partners. Co-authored by Maria Cavaller, our former Patient Engagement and Therapeutic Development Director, the article highlights how strengthening young people's involvement in rare disease research can help ensure that different types of research are meaningful, relevant and reflective of their lived experiences.
Committed to Curing Childhood Blindness
https://savesightnoweurope.org/4th-edition-charity-gala-la-nit-de-la-visio-2026/
Save Sight Now Europe (Fundació Síndrome d'Usher) is hosting the fourth edition of its annual gala, bringing together families, researchers and supporters to raise awareness of Usher syndrome type 1B and raise funds for research towards a treatment for this cause of childhood deafblindness. The evening includes a dinner, a charity auction and live music
Committed to Curing Childhood Blindness
Thousands of children around the world are born with hearing and balance problems due to Usher Syndrome Type 1B. They will also be losing their sight during their childhood years due to the degeneration of the retina caused by Retinitis Pigmentosa.
https://savesightnoweurope.org/
sábado, 17 de octubre de 2026
Clues to what causes multiple sclerosis
Clues to what causes multiple sclerosis
Pointing toward better treatments
Multiple sclerosis is a disease where the body’s immune system attacks the central nervous system. There is no cure, although there are treatments that can reduce the number and severity of relapses. Scientists are studying the disease’s underlying biology to find new clues about its causes and better ways to treat it.
https://www.nih.gov/news-events/nih-research-matters/clues-what-causes-multiple-sclerosis
Breast Cancer
https://www.cancer.gov/types/breast
Breast Cancer
Breast cancer is the second most common cancer in women after skin cancer. Mammograms can detect breast cancer early, possibly before it has spread. Explore the links on this page to learn more about breast cancer prevention, screening, treatment, statistics, research, clinical trials, and more.
From Resection to Dose Individualized Antigen-Directed Therapeutics in Immuno-Oncology CME Phoenix Convention Center Saturday, November 7, 2026 | 12:30 pm - 1:30 pm MT
From Resection to Dose
Individualized Antigen-Directed Therapeutics in Immuno-Oncology CME
Phoenix Convention Center
https://na.eventscloud.com/website/100106/
Saturday, November 7, 2026 | 12:30 pm - 1:30 pm MT
Messenger RNA (mRNA)-based therapeutics have moved from concept into clinical evaluation across immuno-oncology. This symposium discusses how mRNA-encoded, antigen-directed approaches generate anti-tumor immunity, and how they sit alongside checkpoint inhibition, cell therapy and other novel modalities. Faculty will cover the mechanistic basis of mRNA therapies — antigen selection and prediction, delivery, and the resulting T-cell priming and expansion — and how this differs from other immunotherapeutic approaches.
The program will review current clinical evidence across tumor types and disease settings, including earlier-stage and minimal residual disease contexts, matching the strength of each conclusion to the design that produced it. Sessions will also address biomarker and patient-selection considerations, including tumor mutational burden, neoantigen burden and circulating tumor DNA (ctDNA)-based measures, and the multidisciplinary requirements of delivering individualized therapies in practice.
The From Resection to Dose: Individualized Antigen-Directed Therapeutics in Immuno-Oncology, content and views expressed therein are those of Medscape Education and not of SITC.
Translating Molecular Insights Into Practice for HR-Positive/HER2-Negative Metastatic Breast Cancer Authors: Hope Rugo, MD; Peter A. Fasching, MD (++)
Translating Molecular Insights Into Practice for HR-Positive/HER2-Negative Metastatic Breast Cancer
Authors: Hope Rugo, MD; Peter A. Fasching, MD
https://www.medscape.org/viewarticle/translating-molecular-insights-practice-hr-positive-her2-2026a1000y0g?page=1&src=mkmcmr_driv_32025_mscpedu_451770.18_ace_launch-ret&uac=148436CN
Latest Breakthroughs in Breast Care Management: Highlights From Chicago 2026
Authors: Erica L. Mayer, MD, MPH; Michael Untch, MD, PhD; Sherene M. Loi, MD, PhD
https://www.medscape.org/viewarticle/latest-breakthroughs-breast-care-management-highlights-2026a1000kok?page=1&src=mkmcmr_driv_32025_mscpedu_451770.18_ace_launch-ret&uac=148436CN
When the GI Workup Doesn’t Explain the Symptoms Alli Spotts-De Lazzer, MA
https://www.medscape.com/viewarticle/when-gi-workup-doesnt-explain-symptoms-2026a10011tj?ecd=WNL_trdalrt_pos1_ous_261016_etid8801359&uac=148436CN&impID=8801359
Your patient reports ongoing constipation, feeling full after a few bites, bloating, and nausea after meals. You order a gastric emptying study, and it shows delayed emptying. What’s on your differential? A motility disorder? Medication effect? Another gastrointestinal condition?
Kraepelin Disease and Bleuler Disease: Better Terms for Bipolar Disorder, Unipolar Depression, and Schizophrenia? (+++)
My Approach: Discordance Between A1c and GMI
https://www.medscape.com/viewarticle/my-approach-discordance-between-a1c-and-gmi-2026a100127l?ecd=wnl_edit_tpal_etid8803739&uac=148436CN&impID=8803739
Kraepelin Disease and Bleuler Disease: Better Terms for Bipolar Disorder, Unipolar Depression, and Schizophrenia?
https://www.medscape.com/viewarticle/kraepelin-disease-and-bleuler-disease-better-terms-bipolar-2026a100127j?ecd=wnl_edit_tpal_etid8803739&uac=148436CN&impID=8803739
Lessons on Living and Kindness From a Doctor With Cancer
https://www.medscape.com/p04/lessons-living-and-kindness-doctor-cancer-2026a10012fz?ecd=wnl_edit_tpal_etid8803739&uac=148436CN&impID=8803739
Inherited genes could help make CAR T therapies safer and more effective Analysis of two pivotal Yescarta clinical trials revealed that patients' germline DNA influences both toxicity risk and CAR T expansion, opening new opportunities for smarter cell therapy design. Written byBree Foster, PhD
Inherited genes could help make CAR T therapies safer and more effective
Analysis of two pivotal Yescarta clinical trials revealed that patients' germline DNA influences both toxicity risk and CAR T expansion, opening new opportunities for smarter cell therapy design.
Written byBree Foster, PhD
https://www.drugdiscoverynews.com/inherited-genes-could-help-make-car-t-therapies-safer-and-more-effective-17405?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz--OP0iwRY17IhELkfD401G13cRlpE8GxfwbTQ8wqGXvvgUo5oiJu7-Z3VXYca-mtRDx2tjtgr9LiPQMG2YAJFVBBE0nlg&_hsmi=444660716&utm_content=444660716&utm_source=hs_email
Since the first CAR T therapy won approval in 2017, these engineered immune cells have transformed treatment for several blood cancers. However, despite promising responses in many patients, CAR T remains unpredictable. Some patients experience severe cytokine release syndrome (CRS) or neurotoxicity, while others tolerate treatment well. Likewise, the extent to which CAR T cells expand after infusion, a key driver of therapeutic success, varies widely between individuals.
In a study published in Science Immunology, investigators analyzed whole-genome sequencing data from patients enrolled in Kite Pharma's pivotal ZUMA-1 and ZUMA-7 clinical trials of axicabtagene ciloleucel (axi-cel), marketed as Yescarta. Their findings suggest that inherited genetic variants can shape both the safety and performance of CAR T therapy, offering new ways to predict toxicity and engineer improved cell therapies.
A new blueprint for the rational design of siRNA drugs For the first time, researchers imaged a human protein essential to the RNA interference process and found ways to design better siRNA drugs. Written byAllison Whitten, PhD
A new blueprint for the rational design of siRNA drugs
For the first time, researchers imaged a human protein essential to the RNA interference process and found ways to design better siRNA drugs.
Written byAllison Whitten, PhD
https://www.drugdiscoverynews.com/a-new-blueprint-for-the-rational-design-of-sirna-drugs-17296?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-_8FqU5l4UXH7xA7y7xND1dOWXaaGbCkGgQWZPBEmH3TqxmpWDa8zLKC7houKjjgDRqf159BsDlRsXA-8lqFVlXC7Xq6A&_hsmi=444660716&utm_content=444660716&utm_source=hs_email
Small interfering RNA (siRNA) drugs take advantage of RNA interference (RNAi) to “silence” genes with major advantages: They can target any gene without changing its underlying DNA. But designing these drugs has been difficult because so many different sequences of siRNA must be tested to find out which one works best to silence the target. So far, drug developers have been in the dark about why certain siRNA sequences work so much better than others, or how to predict which ones will be successful.
FDA announces new initiative for rare diseases Positive Alzheimer's trial data, workforce reductions, mRNA cancer therapy updates, and more led the news this week. Brought to you byDDN editorial team
FDA announces new initiative for rare diseases
Positive Alzheimer's trial data, workforce reductions, mRNA cancer therapy updates, and more led the news this week.
Brought to you byDDN editorial team
https://www.drugdiscoverynews.com/fda-announces-new-initiative-for-rare-diseases-17591?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-_0WsEXv4_HPLhEO2oRpsUuhBRW69o_BGqi7g3XEGAZaBNoMYKGclt4cnQWrnVNKcn0thGX7v7yN2hY2yoXu7HDEbodVw&_hsmi=444660716&utm_content=444660716&utm_source=hs_email
The FDA has unveiled a new Rare Disease Framework Initiative designed to “reimagine the development of drugs and biologics for rare diseases,” according to the press release. Recognizing that only five percent of rare diseases having an FDA-approved treatment, the announcement describes how the new initiative seeks to include patients’ voices, build shared resources on rare disease drug development and review, and champion the use of cutting-edge science, among other commitments. As outlined in the new white paper accompanying the framework, “The goal is a regulatory framework where fit-for-purpose evidentiary expectations are the norm for all rare disease applications; the FDA will work closely with sponsors and patients to support innovative approaches to generating evidence that will meet the FDA’s rigorous statutory standard of safety and effectiveness.” – Allison Whitten
Beyond CGRP: The Search for Migraine’s Next Big Target Megan Brooks
https://www.medscape.com/viewarticle/beyond-cgrp-search-migraines-next-big-target-2026a100125z?ecd=mkm_ret_261017_mscpmrk_neuro_migraine_etid8804565&uac=148436CN&impID=8804565
For the past decade, the calcitonin gene-related peptide (CGRP) pathway has been a major focus of migraine drug development. The resulting monoclonal antibodies and gepants have transformed clinical practice, establishing CGRP as one of the most successful therapeutic targets in migraine.
Skill Checkup: A Man With Metastatic BRAF Wild-Type Melanoma Treated With Anti-PD-1 Monotherapy Reviewed by Maurie Markman, MD
https://reference.medscape.com/viewarticle/cutaneous-metastatic-melanoma-and-wild-braf-2026a1000ws1?src=&_gl=1*zafw5x*_gcl_au*MTA2Mzc5MDEwMS4xNzg5ODMwNjQxLjIxMjk0OTQzNzYuMTc5MjI0MDIyNS4xNzkyMjQwMzE5LjQyODQzNDEwMi4xNzkyMjQwMjI1LjE3OTIyNDAzMTk.
A 58-year-old White man in the US with a history of fully resected primary cutaneous melanoma of the back returns for follow-up after 10 months of pembrolizumab monotherapy for biopsy-confirmed metastatic disease involving the liver and mediastinal lymph nodes; he had no objective response at any point during the pembrolizumab treatment course. Restaging CT scan shows growth of the known hepatic lesion and mediastinal adenopathy, with no new sites of disease; he reports no new symptoms and remains fully active (Eastern Cooperative Oncology Group [ECOG] performance status 0). Molecular testing performed at the time of metastatic diagnosis showed no BRAF V600E or V600K mutation. He has no history of autoimmune disease and has tolerated pembrolizumab without immune-related toxicity to date. His oncologist confirms radiographic disease progression and discusses transitioning to a new systemic therapy.
Better Conversations, Better Choices: Shared Decision-Making in Prostate Cancer Care Timothy J. Daskivich, MD, MSHPM; Maria P. Mogollon, MD
https://www.medscape.com/viewarticle/better-conversations-better-choices-shared-decision-making-2026a100121a?src=
Shared decision-making (SDM) — the process by which doctors and patients collaboratively arrive at a treatment decision informed by both clinical evidence and the values and preferences of the patient — is a fundamental component of daily urologic practice. Because localized prostate cancer (PC) treatment options offer comparable oncologic outcomes, patients make choices on therapy based on side effects, salvage options, invasiveness, duration of therapy, and their own personal preferences. For this reason, comprehensive and balanced discussion of all these considerations is required for SDM to be successful. Yet, while SDM is universally endorsed by guidelines, execution in clinical practice is often suboptimal, and objective studies have found that physicians spend little time on patient engagement. In this article, we offer insights on how to optimize patient-physician communication for SDM, citing data on how doctors communicate in real-world practice to inform a structure for the conversation and how to avoid common pitfalls.
Cervical Cancer Screening Rates Rise, But Disparities Remain October 16, 2026
https://www.medscape.com/viewarticle/cervical-cancer-screening-rates-rise-disparities-remain-2026a100132u?src=
Cervical cancer screening (CCS) rates increased across all racial and ethnic groups from 2019 to 2024, with Hispanic patients experiencing the largest gain — rising from 9.85% to more than 15%. However, American Indian, Alaska Native, Native Hawaiian, and Pacific Islander patients consistently had the lowest screening rates throughout the study period, and patients in Western and Southern census regions also had lower rates compared with other areas.
The Forgotten Headaches: Too Rare to Care? Dawn Attride October 12, 2026
https://www.medscape.com/viewarticle/forgotten-headaches-too-rare-care-2026a100126b?ecd=mkm_ret_261017_mscpmrk_neuro_migraine_etid8804565&uac=148436CN&impID=8804565
For headache neurologist Christopher Gottschalk, MD, severe pain is familiar territory, but cluster headache stands apart — not only for the agony it inflicts but for the gulf between patient suffering and what medicine has to offer.
viernes, 16 de octubre de 2026
Newborn screening across EU needs to become more equitable
https://www.healthawareness.co.uk/rare-diseases/newborn-screening-across-eu-needs-to-become-more-equitable/
Our Research and Policy Senior Manager, Gulcin Gumus, has written for Mediaplanet about inequalities in newborn screening across the EU. She explains how access to early diagnosis and care can depend on where a child is born, and calls for stronger EU-level collaboration to ensure more equitable access to newborn screening across Member States.
ERNs on Air
https://open.spotify.com/episode/2VihltLPdeb7EHViyEANf4
ERNs on Air
The latest episode of European Reference Networks (ERNs) on Air, released on 5 October, is hosted by Nora Lázaro, Patient Engagement Manager for ERNs and Healthcare at EURORDIS. In this episode, Nora discusses the patient-led initiative of the Sudden Cardiac Death Letter for Families with Ruth Biller, ePAG Lead in ERN GUARD-Heart, and Lia Crotti, a cardiologist in the network. Together, they discuss how the lived experiences of people affected by rare diseases helped identify an unmet need and highlight the importance of putting patient partnership into practice across ERNs
10 Minutes With Rhiannon Walls: Youth Leadership
https://rememberthegirls.org/
10 Minutes With...
Catch up on our latest episode of 10 Minutes With..., in which our colleague Rhiannon Walls speaks with Taylor Kane, founder and Executive Director of Remember The Girls. Taylor Kane's advocacy journey began in childhood, shaped by her own and her family's experience of adrenoleukodystrophy (ALD). Their conversation highlights the importance of taking young advocates seriously, listening to their lived experiences and empowering them to turn those experiences into meaningful action.
https://www.eurordis.org/10-minutes-with-rhiannon-walls/
13th European Conference on Rare Diseases and Orphan Products (ECRD 2026). ECRD 2026,
https://www.rare-diseases.eu/previous-ecrds/
'Rare Diseases in a Changing and Competitive Europe, shaping policies to address the unmet needs of people living with rare diseases, hybrid ECRD''
ECRD 2026
EURORDIS recently published the Executive Summary of the 13th European Conference on Rare Diseases and Orphan Products (ECRD 2026). ECRD 2026, which took place in June, marked a key milestone in the inclusive, multi-stakeholder process contributing to the development of the now-launched European Blueprint for Rare Diseases.
https://www.rare-diseases.eu/
Health for a stronger European Union High time for action! (28 videos)
https://www.ehfg.org/
EHFG 2026
Our CEO Jean Saslawsky and our Public Affairs Director Rosa Castro recently attended the European Health Forum Gastein 2026. The conference focused on strengthening European health systems and cross-border collaboration amid growing political uncertainty. Jean and Rosa highlighted the importance of advancing a European Action Plan for Rare Diseases and ensuring patient involvement in the development of regulatory sandboxes
https://www.youtube.com/playlist?list=PLYwFW4aHI1vA
Webinar Series: Kickstart your advocacy: Practical Advocacy: demystifying European institutions Fecha y hora 26 nov 2026 12:00
https://www.eurordis.org/webinar-series-kickstart-your-advocacy/
Kickstart Your Advocacy
This autumn, we are running a series of webinars that recognise the importance of young people in shaping the future of the rare disease community. The third webinar takes place online on 26 November and explores how EU institutions and legislative processes work, helping young people understand how and where they can engage in European advocacy. Register now! This webinar series has been exclusively supported by the Stavros Niarchos Foundation (SNF) through its 30 for 30: Next-Gen Grants Initiative.
This series of four free webinars for young people under 30 years old in the rare disease community is designed to provide practical tools, knowledge and skills to help young people strengthen their advocacy and create meaningful change.
Whether you are living with a rare disease, supporting someone who is, or already established in rare disease advocacy, the series will explore four key areas: communication and storytelling, leadership, European advocacy, and mental wellbeing and resilience.
https://www.snf.org/
Practical Advocacy: demystifying European institutions
Fecha y hora
26 nov 2026 12:00
https://us02web.zoom.us/webinar/register/WN_1IAwrEy7Tgm4Vm9NjfmtTw#/registration
Rare Disease Day 2027: Launch Webinar
https://www.rarediseaseday.org/webinars/
Rare Disease Day
Interested in being involved in the Rare Disease Day 2027 campaign, ahead of 28 February 2027? To find out how you can get involved and how we are preparing for it, join one of the two webinars taking place on Monday 19 October at 16:00 CEST and Tuesday 20 October at 09:00 CEST. There is no need to attend both sessions, as the same information will be covered in each.
Rare Disease Day 2027
Fecha y hora
19 oct 2026 11:00
https://us02web.zoom.us/webinar/register/WN_Ck2iiRCAQZe4d5Cr9EYYfA#/registration
Rare Disease Day 2027
Fecha y hora
20 oct 2026 04:00
https://us02web.zoom.us/webinar/register/WN_OwOo8lGNQxqji5wtINbV2g#/registration
Patient-Centred Survey on Clinical Trials.
https://survey.erknet.org/#/s/kjcpbfqqtr
Patient survey
On 12 October, the IHI RealiseD project, to which EURORDIS contributes, launched its Patient-Centred Survey on Clinical Trials. The survey aims to better understand the barriers, needs and priorities of people living with rare diseases and their families and carers. By sharing their experiences, respondents can help make rare disease clinical research more accessible, inclusive and patient-centred. Take the survey now.
Advancing Clinical Trials for Rare and Ultra-Rare Diseases
RealiseD is set to generate a novel comprehensive methodological and operational approach to design and conduct clinical trials for rare and ultra-rare diseases.
https://realised-ihi.eu/
Expert Dialogue on the EU Biotech Act
EU Biotech Act
Earlier this week, the Intergroup also convened an expert dialogue with MEP Stine Bosse to discuss the proposed Biotech Act and explore how it could strengthen clinical research, foster innovation and improve treatment access. Our CEO, Jean Saslawsky, stressed the importance of supporting non-profit developers, building on existing European research infrastructure and recognising patients as equal partners in innovation
Fostering European political unity for cancer and rare diseases: Expert Dialogue on Cross-Border Healthcare Wednesday, November 4, 2026 9:30 AM 11:30 AM
Fostering European political unity for cancer and rare diseases
The European Parliament Intergroup on Cancer and Rare Diseases hosted its constitutive meeting on 4 February 2025 at the European Parliament in Brussels
https://www.intergrouponcancerandrarediseases.eu/
Cross-border healthcare
On 4 November, the European Parliament Intergroup on Cancer and Rare Diseases will organise an expert dialogue exploring how Europe's cross-border healthcare framework can better meet the needs of people living with cancer and rare diseases. Registration to attend is open until 22 October. The discussion will examine barriers to accessing healthcare across borders and how modernised EU rules could improve access to specialised care, clinical trials and treatment, regardless of where patients live.
Expert Dialogue on Cross-Border Healthcare
Wednesday, November 4, 2026
9:30 AM 11:30 AM
https://www.intergrouponcancerandrarediseases.eu/events/expert-dialogue-on-cross-border-healthcare
Europe’s rare disease community sets out Blueprint for coordinated EU action
https://www.eurordis.org/europes-rare-disease-community-sets-out-blueprint-for-coordinated-eu-action/
EURORDIS has today launched its European Blueprint for Rare Diseases, setting out the rare disease community's proposals for the priorities and delivery of a future EU Action Plan for Rare Diseases.
The Blueprint aims to address existing gaps, strengthen coordination and improve outcomes across Europe.
Developed over the past 10 months, the Blueprint is the result of a collaborative effort involving people living with rare diseases, patient organisations, clinicians, researchers, public authorities and industry, all working towards a shared vision for coordinated EU action.
EURORDIS is calling on the European Commission to propose an EU Action Plan for Rare Diseases, using the Blueprint as the rare disease community's contribution to its development.
With more than 95% of rare diseases lacking treatment, diagnoses taking an average of six years and significant inequalities persisting across EU Member States, the time for such an Action Plan is now. The Blueprint represents a major step towards achieving this goal.
FDA Approves New Nonprescription Eye Drop for Relief of Eye Redness
https://www.fda.gov/drugs/drug-alerts-and-statements/fda-approves-new-nonprescription-eye-drop-relief-eye-redness?utm_medium=email&utm_source=govdelivery
Today, the U.S. Food and Drug Administration approved a new nonprescription eye drop treatment, Brytly, apraclonidine ophthalmic solution, 0.125%, for relief of eye redness due to minor irritations. This is the first nonprescription eye drop with apraclonidine.
This approval aligns with FDA’s Public Health Pillars, which prioritize increasing access to affordable medicines. The agency is actively working to help foster conditions that encourage competition and expand the availability of nonprescription drugs.
Eye redness is a common condition most frequently caused by swollen or dilated blood vessels on the surface of the eye, which can result from minor irritations such as dryness or dust. Eye drops with higher concentrations of apraclonidine are available by prescription to reduce eye pressure in patients with glaucoma or who are undergoing certain types of laser eye surgery.
Brytly is approved only for adults and children 12 years of age and older and should not be used more than two times per day.
Before using Brytly, consumers should read the full Drug Facts label. Consumers should remove contact lenses before use and wait at least 10 minutes after applying the eye drops before re-inserting contact lenses. If additional eye drops will be used, consumers should wait five minutes between applying each product. Consumers should stop using Brytly and ask a doctor if they experience eye pain, changes in vision, continued redness, or irritation or if the condition persists for more than three days or worsens.
An inherited hypomorphic variant in PIEZO2 reveals structural features of mechanotransduction (+++)
https://pmc.ncbi.nlm.nih.gov/articles/PMC13588269/
New NCCIH Finding on PIEZO2 Advances Understanding of Touch and Proprioception
A newly identified genetic change in the PIEZO2 gene has revealed a critical piece of how the body senses touch, movement, and position, according to a new study based on research conducted by the Sensory Cells and Circuits Lab at the National Center for Complementary and Integrative Health (NCCIH) and recently published in the journal Neuron.
https://www.nccih.nih.gov/research/intramural/intramural-labs/sensory-cells-and-circuits-lab/about
Researchers found that a single amino acid change can alter the function of the PIEZO2 protein and impair its ability to respond to mechanical force, helping explain a rare neurological condition in two fraternal twins who experience balance problems, impaired body-position sensing, and scoliosis. With this single amino acid change, PIEZO2 proteins, which sit in the outer membrane of cells and function as channels for ions to go through, appear to be locked in a closed state and unable to transition to opening.
https://pubmed.ncbi.nlm.nih.gov/42636812/
The discovery adds insight to how the PIEZO2 protein converts physical force into nerve signals and could help guide future research into disorders affecting touch, proprioception, and other sensory functions that depend on PIEZO2 proteins.
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