The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...
Columnist Betty Vertin wishes she could take away the pain over the milestones in the teenage years her sons with DMD will miss.
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