The US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) have voted eight to six in favour of a new gene therapy for Duchenne muscular dystrophy. The vote supports the accelerated approval of Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with Duchenne muscular dystrophy with a […]
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