CIENCIASMEDICASNEWS

martes, 22 de septiembre de 2026

Trump says he’ll require drugmakers to end multiple vaccine doses, reviving discredited autism claims Washington Post | September 22, 2026

https://geneticliteracyproject.org/2026/09/22/trump-says-hell-require-drugmakers-to-end-multiple-vaccine-doses-reviving-discredited-autism-claims/ President Trump said that he’ll “demand” drugmakers break vaccines into five smaller doses spaced six months apart, claiming the change would produce a “massive reduction in autism,” as the Washington Post’s Rachel Roubein reports. It wasn’t immediately clear which vaccines he had in mind.

Viewpoint: Study links childhood vaccination to autism!!! Here’s how anti-vaxxers twist evidence to corrupt science Charles Dinerstein | September 22, 2026

https://geneticliteracyproject.org/2026/09/22/viewpoint-study-links-childhood-vaccination-to-autism-heres-how-anti-vaxxers-twist-evidence-to-corrupt-science/ Aviral post on X claims the “largest vaccinated vs. unvaccinated birth cohort study ever” found vaccinated children far sicker across every category of chronic disease. The numbers sound alarming, but follow them back to their source, and the story becomes less about vaccines and more about scientific uncertainty transformed into social-media certainty.

Prioritising pain management in inflammatory arthritis The Lancet Rheumatology ++... ++

Prioritising pain management in inflammatory arthritis The Lancet Rheumatology https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(26)00286-9/fulltext?dgcid=raven_jbs_etoc_email Oct 2026 Volume 8Number 10e757-e828 https://www.thelancet.com/journals/lanrhe/issue/vol8no10/PIIS2665-9913(26)X2009-4 Diagnostic accuracy of musculoskeletal ultrasound in suspected polymyalgia rheumatica: a multicentre, cohort study Christoffer Søvsø Våben, MDa,c Send email to chrmoa@rm.dk ∙ Andreas Wiggers Nielsen, MD PhDa,b,c ∙ Maria Sandovici, MD PhDd ∙ Søren Geill Kjær, MDe ∙ Ib Tønder Hansen, MD PhDa ∙ Jesper Blegvad-Nissen, MDe ∙ et al. https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(26)00187-6/abstract?dgcid=raven_jbs_etoc_feature_lanrhe Defining age-related thresholds in hand, wrist, and foot ultrasound: an international, cross-sectional, observational study by the Outcome Measures in Rheumatology Ultrasound Working Group Ilfita Sahbudin, BM PhDa,b ∙ Jeanette Trickey, MDb,c ∙ Hélène Gouze, MD PhDd ∙ Prof Maria Simona Stoenoiu, MD PhDe ∙ Georgios Filippou, MD PhDf,g ∙ Garifallia Sakellariou, MD PhDh,i ∙ et al. https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(26)00183-9/abstract?dgcid=raven_jbs_etoc_feature_lanrhe

Zilganersen for the Treatment of Alexander Disease

Amy Waldman, MD, pediatric neurologist and Medical Director of the Leukodystrophy Center at the Children’s Hospital of Philadelphia, discusses the approval of zilganersen to treat patients with Alexander disease. Alexander disease is a rare leukodystrophy that impairs neuronal signalling. Most cases of Alexander disease begin before age 2 and are described as the infantile form. Signs and symptoms of the infantile form typically include an enlarged brain and head size, seizures, stiffness in the arms and/or legs, intellectual disability, and developmental delay. Alexander disease is also characterized by abnormal protein deposits known as Rosenthal fibers. These deposits are found in specialized cells called astroglial cells, which support and nourish other cells in the central nervous system. Zilganersen is an antisense oligonucleotide that binds to RNA and is designed to inhibit production of excess glial fibrillary acidic protein (GFAP) that accumulates as a result of pathogenic variants in the GFAP gene. It is the first and only disease modifying therapy approved for this indication. The approval is based on positive results from a global, multicenter, randomized, double-blind, controlled, multiple-ascending dose (MAD) phase 1-3 study (NCT04849741) that enrolled 54 participants with Alexander disease. The study met its primary endpoint in individuals 5 years of age and older, with zilganersen 50 mg demonstrating statistically significant and clinically meaningful stabilization of gait speed as assessed by the 10-Meter Walk Test (10MWT) compared to control at week 61. The treatment also demonstrated a favorable safety and tolerability profile, with most adverse events being mild or moderate in severity. Alexander disease faces a unique challenge to getting on newborn screening panels. Although the disease does typically present in young children, GFAP accumulation may not be high enough to detect at birth. Dr. Waldman explains that research is still being done to determine when the earliest possible diagnosis can be made. However, she highlights the following red flag symptoms that may point to central nervous system involvement and may warrant genetic testing: in younger children these include gross motor and developmental delays, seizures, and in older children, these include more mild symptoms such as hypernasal speech, speech delay, vomiting, and gait imbalance. For more information on the approval, visit https://ir.ionis.com/news-releases/ne... To learn more about Alexander disease and other rare neurological conditions, visit https://checkrare.com/diseases/neurol...

Topline Results From the SANRECO Trial Testing Divesiran in Polycythemia Vera by Madaline Spencer| Published on: Sep 21, 2026

Topline Results From the SANRECO Trial Testing Divesiran in Polycythemia Vera by Madaline Spencer| Published on: Sep 21, 2026 Marina Kremyanskaya, MD, PhD, Associate Professor at Icahn School of Medicine at Mount Sinai, discusses the phase 2 topline findings from the SANRECO clinical trial and the evolving polycythemia vera (PV) treatment landscape. https://checkrare.com/topline-results-from-the-sanreco-trial-testing-divesiran-in-polycythemia-vera/

What to Expect: 2026 MGFA Scientific Session by Madaline Spencer| Published on: Sep 22, 2026

What to Expect: 2026 MGFA Scientific Session by Madaline Spencer| Published on: Sep 22, 2026 Jenny McCue, Vice President of Global Research and Clinical Development at the Myasthenia Gravis Foundation of America (MGFA), discusses research highlights to be presented at the upcoming 2026 MGFA Scientific Session being held September 29th in Orlando, FL. https://checkrare.com/what-to-expect-2026-mgfa-scientific-session/