Considering New Duchenne Muscular Dystrophy Therapies: Excon Skipping and Gene Therapy

PBGENE-DMD therapy gets FDA's rare pediatric disease status

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect children. “The receipt of Rare Pediatric Disease Designation highlights the significan…

Considering New Duchenne Muscular Dystrophy Therapies: Excon Skipping and Gene Therapy

A panelist discusses how older DMD patients might benefit from newer genetic therapies including gene therapy and exon skipping, though eligibility depends on specific mutations and absence of pre-existing immunity to AAV vectors.