recombinant adeno-associated virus serotype rhesus 74 (rh74) expressing the human CT GalNac transferase (GALGT2) gene, also called the Sda or Cad GAlNac transferase or b4Galnt2, under control of the muscle creatine kinase (MCK) promoter

recombinant adeno-associated virus serotype rhesus 74 (rh74) expressing the human CT GalNac transferase (GALGT2) gene, also called the Sda or Cad GAlNac transferase or b4Galnt2, under control of the muscle creatine kinase (MCK) promoter
Trade Name
Orphan Indication Duchenne muscular dystrophy
USA Market Approval USA
USA Designation Date 2015-06-16 00:00:00
Sponsor Nationwide Children's Hospital;700 Children's Drive, Rm WA2025;Columbus, Ohio, 43205
Related Access Program
PTC Therapeutics – Duchenne Muscular Dystrophy Marathon Pharmaceuticals, LLC. – Duchenne muscular dystrophy PTC Therapeutics International Limited – Duchenne muscular dystrophy