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PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
duchenne muscular dystrophy with mutations amenable to pbgene-dmd, duchenne muscular dystrophy, dmd, x-linked muscle wasting disease, muscular dystrophy, progressive muscle weakness, gene editing, aav serotype 9, aav-9, exon 45-55 excision, interventional, phase1
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