BioMarin Pharmaceutical: Muscular Dystrophy, Duchenne trials

The registry lists 10 Muscular Dystrophy, Duchenne studies with BioMarin Pharmaceutical as lead sponsor. 1 are open to participants now and 1 have results posted.

Phase 1 4Phase 2 7Phase 3 1No phase 2(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular DystrophyEnrolling by invitationNCT07573631Phase 2
• Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)CompletedNCT01037309Phase 1/2Results posted
• A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular DystrophyActive, not recruitingNCT06280209Phase 1/2
• Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)TerminatedNCT02958202Phase 2
• Extension Study of Drisapersen in DMD SubjectsNo longer availableNCT02636686
• Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)TerminatedNCT02329769Phase 2
• A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)TerminatedNCT01957059Phase 1/2
• Phase IIb Study of PRO045 in Subjects With Duchenne Muscular DystrophyTerminatedNCT01826474Phase 1/2
• A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian SubjectsTerminatedNCT01803412Phase 3
• A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.TerminatedNCT01753804