Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2007-05-01
Target enrollment:
Participant gender:
Summary
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense
mutation (premature stop codon) in the gene that makes the dystrophin protein. PTC124 has
been shown to partially restore dystrophin production in animals with DMD due to a nonsense
mutation. The main purpose of this study is to understand whether PTC124 can safely increase
functional dystrophin protein in the muscles of patients with DMD due to a nonsense mutation.