FDA Accepts BridgeBio’s NDA for BBP‑418 as Potential First Limb‑Girdle Muscular Dystrophy Treatment on May 28, 2026 BBP-418 BridgeBio Clinical Trials FORTIFY LGMD2I/R9 Muscular Dystrophies, Limb-Girdle NDA PDUFA Priority Review United States Food and Drug Administration +
Untangling Sarepta's Gene Therapy Fallout and Trust Deficit on August 04, 2025 AAVrh74 vector clinical holds Elevidys gene therapy Muscular Dystrophies, Limb-Girdle Muscular Dystrophy, Duchenne patient deaths Regulatory Action Translucent United States Food and Drug Administration +
Lack of Transparency Tarnishes Sarepta's Sheen as Patient Deaths Trigger FDA Battle on July 23, 2025 Elevidys gene therapy lack of transparency Market Muscular Dystrophies, Limb-Girdle Muscular Dystrophy, Duchenne patient deaths Regulatory Action United States Food and Drug Administration Withdraw (activity) +
Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy on July 18, 2025 AAVS1 gene Elevidys gene therapy Liver Failure, Acute Muscular Dystrophies, Limb-Girdle Patient Death Safety Study on Hold United States Food and Drug Administration vector +
Sarepta's rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy on June 04, 2025 gene therapy Infrequent Muscular Dystrophies, Limb-Girdle platform technology designation SRP-9003 United States Food and Drug Administration Viral Vector +