Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene

Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
Trade Name
Orphan Indication Duchenne muscular dystrophy
EU Market Approval EU
EU Designation Date 2016-08-29 00:00:00
Sponsor Pfizer Limited
Related Access Program
PTC Therapeutics – Duchenne Muscular Dystrophy Marathon Pharmaceuticals, LLC. – Duchenne muscular dystrophy PTC Therapeutics International Limited – Duchenne muscular dystrophy