ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952 ·Status: COMPLETED ·Phase: PHASE1

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

NCT05230459 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Study of Eteplirsen in DMD Patients

NCT02255552 ·Status: COMPLETED ·Phase: PHASE3

Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III

NCT06971094 ·Status: RECRUITING ·Phase: PHASE3

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

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437 ·Status: UNKNOWN ·Phase: PHASE1

Treatment of a Single Patient With CRD-TMH-001

NCT05514249 ·Status: UNKNOWN ·Phase: PHASE1

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372 ·Status: TERMINATED ·Phase: PHASE2

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

NCT07070999 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

NCT03218995 ·Status: COMPLETED ·Phase: PHASE2

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05096221 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)

NCT03375255 ·Status: COMPLETED ·Phase: PHASE1

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

NCT05824169 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

NCT01344798 ·Status: COMPLETED ·Phase: PHASE1

A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.

NCT06392724 ·Status: ACTIVE_NOT_RECRUITING ·Phase: EARLY_PHASE1

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

NCT06128564 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

NCT06421831 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00592553 ·Status: COMPLETED ·Phase: PHASE2

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

NCT05775848 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

NCT02819557 ·Status: COMPLETED ·Phase: PHASE2

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631 ·Status: COMPLETED ·Phase: PHASE3

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

NCT00494195 ·Status: COMPLETED ·Phase: PHASE1

Study of ATH434 in Participants with Multiple System Atrophy

NCT05109091 ·Status: COMPLETED ·Phase: PHASE2