source
- Opinion: Limb girdle muscular dystrophy patients face a maddening reality“Too often, regulatory tools authorized to speed patient access to breakthrough treatments are being used in ways that even prevent approval,” writes Kat Bryant Knudson.
- FDA panel rejects Capricor's Duchenne drug, citing insufficient efficacy dataThe U.S. Food and Drug Administration advisory committee voted 9-to-3 against a Capricor Therapeutics candidate for Duchenne muscular dystrophy. The panel said the clinical data did not provide sub…