AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of functional dystrophin, beginning at birth and leading to progressive impaired motor function, loss of ambulation and life-threatening cardiorespiratory complications. Delandistrogene moxeparvovec, an adeno-associated rh74-viral vector-based gene therapy, addresses absent functional dystrophin in DMD. Here the phase 3 EMBARK study aimed to assess the efficacy and safety of delandistrogene moxeparvovec in patients with DMD. Ambulatory males with DMD, ≥4 years to

• P
  Pfizer
• R
  Roche
• MD
  Muscular Dystrophy Association
• B
  Biogen
• PP
  Parent Project Muscular Dystrophy
• F
  FibroGen
• FH
  F. Hoffmann-La Roche
• NC
  Nationwide Children's Hospital
• CB
  CSL Behring
• TP
  Taiho Pharmaceutical
• PT
  PTC Therapeutics
• A
  AveXis
• ST
  Sarepta Therapeutics
• NS
  Nippon Shinyaku
• AP
  Astellas Pharma
• I
  Italfarmaco
• G
  Genentech
• CP
  Chugai Pharmaceutical
• UP
  UCB Pharma