Pathway Development Consortium Announces Publication in Human Gene Therapy on the Application of FDA’s Accelerated Approval Pathway for AAV Gene Therapies for Patients with Duchenne Muscular Dystrophy
– Publication in Human Gene Therapy provides rationale for the use of microdystrophin expression levels as a surrogate endpoint reasonably likely to predict clinical benefit for AAV gene therapy intended for treatment of patients with Duchenne muscular dystrophy –
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