Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876 · clinicaltrials.gov ↗
PHASE2
Phase
TERMINATED
Status
36
Enrollment
INDUSTRY
Sponsor class

Stopped Terminated early because similar study with Ataluren (PTC124-GD-007-DMD; NCT00592553) exhibited lack of efficacy at the high dose (not due to safety concerns).

Conditions

Interventions

Sponsor

PTC Therapeutics

Collaborators