A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I

NCT06576388 ·Status: COMPLETED ·Phase: EARLY_PHASE1

A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA

NCT06588803 ·Status: ENROLLING_BY_INVITATION

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

NCT05901987 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

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

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy

NCT06888661 ·Status: ENROLLING_BY_INVITATION ·Phase: EARLY_PHASE1

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

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

NCT07265232 ·Status: RECRUITING ·Phase: PHASE3

Pediatric Spinal Muscular Atrophy (SMA) China Registry

NCT05042921 ·Status: ACTIVE_NOT_RECRUITING

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

NCT01344798 ·Status: COMPLETED ·Phase: PHASE1

Antisense Oligonucleotide for Spinal Muscular Atrophy

NCT05187260 ·Status: UNKNOWN

Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study

NCT05839145 ·Status: NOT_YET_RECRUITING ·Phase: NA

Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System

NCT07223489 ·Status: RECRUITING

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

NCT05335876 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952 ·Status: COMPLETED ·Phase: PHASE1

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

NCT05517603 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Adult Spinal Muscular Atrophy (SMA) China Registry

NCT05618379 ·Status: ACTIVE_NOT_RECRUITING

Clinical Safety Evaluation and Preliminary Efficacy Study of Subcutaneous Myografts Transplantation

NCT07426068 ·Status: NOT_YET_RECRUITING ·Phase: EARLY_PHASE1

Prospective Follow-up of Patients With Glycogen Storage Disease Type III

NCT01563705 ·Status: UNKNOWN ·Phase: NA

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

NCT07070999 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System

NCT00155168 ·Status: UNKNOWN

Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy

NCT06562283 ·Status: RECRUITING ·Phase: NA

Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

NCT06532474 ·Status: RECRUITING

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

NCT05337553 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

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

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

NCT06321965 ·Status: RECRUITING ·Phase: NA