An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

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

Pediatric Spinal Muscular Atrophy (SMA) China Registry

NCT05042921 ·Status: ACTIVE_NOT_RECRUITING

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03306277 ·Status: COMPLETED ·Phase: PHASE3

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952 ·Status: COMPLETED ·Phase: PHASE1

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

NCT05614531 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE1/PHASE2

Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03461289 ·Status: COMPLETED ·Phase: PHASE3

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

NCT01344798 ·Status: COMPLETED ·Phase: PHASE1

Adult Spinal Muscular Atrophy (SMA) China Registry

NCT05618379 ·Status: ACTIVE_NOT_RECRUITING

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

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

NCT03837184 ·Status: COMPLETED ·Phase: PHASE3

A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)

NCT04729907 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Antisense Oligonucleotide for Spinal Muscular Atrophy

NCT05187260 ·Status: UNKNOWN

GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

NCT06772402 ·Status: ENROLLING_BY_INVITATION ·Phase: NA

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

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

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

NCT05335876 ·Status: RECRUITING ·Phase: PHASE3

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

NCT03505099 ·Status: COMPLETED ·Phase: PHASE3

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05693142 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

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

NCT07070999 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

NCT02550691 ·Status: TERMINATED ·Phase: NA

Measuring Levels of SMN in Blood Samples of SMA Patients

NCT00061607 ·Status: COMPLETED

Adults With SMA Treated With Nusinersen

NCT04591678 ·Status: COMPLETED

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

NCT05839145 ·Status: NOT_YET_RECRUITING ·Phase: NA

MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy

NCT05769465 ·Status: RECRUITING