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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 ...
Annual event brings together 1,200 plus families, physicians, researchers, caregivers, industry partners, and those living with Duchenne and Becke ...
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 ...
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MarketBeat on MSNSarepta Drops 42% on Fatalities; Markets Eye Solid BiosciencesIn another tragic incident, Sarepta Therapeutics (NASDAQ: SRPT) saw a second death in connection with its Duchenne muscular ...
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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