- DMD (Pro053)
- PRO045 (DMD)
- DMD-Heart protection trial (DMD without echo-detectable left ventricular dysfunction)
- Ataluren (PTC124-GD-019) (Nonsense mutation dystrophinopathy)
- Ataluren (PTC124-GD-020) (Nonsense mutation dystrophinopathy)
- SMT C11003 (Duchenne Muscular Dystrophy (DMD))
- BYM338 (Sporadic Inclusion Body Myositis (sIBM))