Musculoskeletal Diseases clinical trials at UC Health
2 in progress, 1 open to new patients

• Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

  open to eligible males ages 5 years and up

  This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

  at UCSF UC Davis

• Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

  Sorry, in progress, not accepting new patients

  The main goal of this Phase 3 extension study is to obtain long term safety of ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and laboratory abnormalities. The study will also assess changes in physical function, pulmonary function and other important clinical and laboratory measures.

  at UCLA UC Davis

Last updated: October 23, 2018