Active, But Not Enrolling Clinical Trials

A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated with Epidiolex (Cannabidiol) Oral Solution (GWEP19022)

A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy (CANYON)

A Phase 3 Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (FORTIFY)

A Phase II Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants with Facioscapulohumeral Muscular Dystrophy

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

Neuronal Ceroid Lipofuscinosis (NCL) Natural History Study

Rare Disease Clinical Research Network Brittle Bone Disease Consortium Longitudinal Study of Osteogenesis Imperfecta (RDCRN #7701)

Ultragenyx Study to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta