After disaster, a rare disease community gets new chance at treatment

Astellas renews trials of gene therapy for XLMTM, hoping for success stories like JJ’s. ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏  ͏ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­ ­  

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Gonzalez Family STAT+ | Five years after disaster, a rare disease community gets new chance at treatment Astellas renews trials on a gene therapy for a fatal muscle-wasting disease, X-linked myotubular myopathy (XLMTM), five years after a disastrous start. By Jason Mast STAT+ | Medicare is spending far less than expected on new Alzheimer’s drugs Feds expected to spend billions, not millions, on Leqembi and Kisunla, but the treatments are complicated to administer and adoption has been slow. By Bob Herman No FDA permission, no problem: New flavored vape policy worries experts FDA announced new enforcement guidance, allowing some unauthorized vapes to stay on the market while applications are processed. Experts foresee problems. By Sarah Todd Camille MacMillin/STAT Animal skin disease confirmed in clusters of European men who have sex with men Doctors in France and Spain report clusters of a rare animal-linked skin infection among men having sex with men (MSM) with no known animal exposure. By Helen Branswell STAT+ | Trump pivots on kratom derivative 7-OH, floating approval for some forms The president appears to favor giving some forms of kratom, a natural opioid, approval, despite recent FDA warnings about such products. By Lev Facher STAT+ | Provider, insurer groups rush to shape No Surprises Act arbitration rules New rules expected soon for the No Surprises Act’s controversial arbitration process, long a source of consternation for providers and insurers alike. By Tara Bannow Adobe Opinion: What addiction medicine can teach us about depending on AI When people hear the word “addiction,” they often assume it implies catastrophe. But it starts with the gradual shift from optional use to psychological reliance. By Jonathan Avery Opinion: AI doctors should be licensed. Here’s a framework to do that Just like doctors, autonomous medical AI should demonstrate competency before practicing, through testing, supervised deployment, and ongoing monitoring. By Alon Bergman

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