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Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...
Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...
Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...
The boys live in Reed City with their mother Tabatha; father Michael, who is the Osceola County Road Commission manager and ...
U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s ...
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...
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Why Sarepta Therapeutics (SRPT) Stock Is Down TodayWhat Happened? Shares of biotech company Sarepta Therapeutics (NASDAQ:SRPT) fell 44.4% in the afternoon session after the ...
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its 2025 Annual Conference in Las Vegas, Nevada, June ...
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