Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
Stopped Terminated early because a similar study with Ataluren exhibited lack of efficacy at the high dose (not due to safety concerns).
Conditions
- Duchenne Muscular Dystrophy
- Becker Muscular Dystrophy
Interventions
- DRUG: Ataluren
- DRUG: Chronic Corticosteroid Therapy
Sponsor
PTC Therapeutics
Collaborators