The FDA and Gene Therapy for Duchenne Muscular Dystrophy
David M. Rind
Abstract
This Viewpoint examines the appropriateness of FDA accelerated approval of novel gene therapies to treat boys with Duchenne muscular dystrophy following clinical trials with surrogate outcomes that did not demonstrate net benefits.
Topics & Concepts
MedicineDuchenne muscular dystrophyClinical trialMuscular dystrophyGenetic enhancementPhysical therapyPhysical medicine and rehabilitationGeneInternal medicineGeneticsBiologyMuscle Physiology and DisordersNeurogenetic and Muscular Disorders ResearchCAR-T cell therapy research