Commission
European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment
Translarna, Duchenne muscular dystrophy, European Commission, EMA, CHMP, conditional marketing authorization, drug approval, regulatory decisions
Biogen and Ionis’ ALS Treatment Qalsody Receives European Commission Approval
Biogen, Ionis, Qalsody, ALS, European Commission, Marketing Authorization, Rare Disease, SOD1-ALS, Neurofilament, Clinical Trials
Sarepta’s ELEVIDYS Gaining Ground Amidst Anticipation for Expanded Label Approval
ELEVIDYS, Age, Muscular Dystrophy, Duchenne, Sarepta, Labels (device), Child
Sarepta shares PhII data for next-gen exon skipping drug, but regulatory pathway is unclear
Regulatory Pathway, Sarepta, next-generation exon-skipping drug, United States Food and Drug Administration, Approved, Muscular Dystrophy, Duchenne, gene therapy, Sarepta’s gene therapy