Long Term Follow-up for RGX-202

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

NCT00428935 ·Status: COMPLETED ·Phase: PHASE1

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

NCT05689164 ·Status: TERMINATED ·Phase: PHASE3

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

NCT03362502 ·Status: TERMINATED ·Phase: PHASE1

An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)

NCT06594094 ·Status: COMPLETED ·Phase: EARLY_PHASE1

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT03368742 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01462292 ·Status: COMPLETED ·Phase: PHASE2

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

NCT06270719 ·Status: ENROLLING_BY_INVITATION

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278 ·Status: COMPLETED

Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

NCT00494195 ·Status: COMPLETED ·Phase: PHASE1

Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy

NCT06900049 ·Status: RECRUITING ·Phase: EARLY_PHASE1

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

NCT02667483 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01957059 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

NCT03508947 ·Status: COMPLETED ·Phase: PHASE1

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02579239 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

NCT06191354 ·Status: ACTIVE_NOT_RECRUITING ·Phase: NA

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02740972 ·Status: COMPLETED ·Phase: PHASE2

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

NCT02420379 ·Status: COMPLETED ·Phase: PHASE2

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

NCT02515669 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Study of ALS Reversals 2: Genetic Analyses

NCT03464903 ·Status: COMPLETED

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)

NCT05906251 ·Status: TERMINATED ·Phase: PHASE1

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947 ·Status: COMPLETED ·Phase: PHASE2

A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT06441682 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

NCT00468832 ·Status: UNKNOWN

Muscle Tissue Bank for Muscular Dystrophy

NCT01950897 ·Status: COMPLETED