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

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

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

NCT06772402 ·Status: ENROLLING_BY_INVITATION ·Phase: NA

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

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

NCT06288230 ·Status: 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

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

NCT07070999 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

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

NCT03306277 ·Status: COMPLETED ·Phase: PHASE3

Natural History of Types 2 and 3 SMA in Taiwan

NCT03300869 ·Status: UNKNOWN

Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System

NCT00155168 ·Status: UNKNOWN

Pediatric Spinal Muscular Atrophy (SMA) China Registry

NCT05042921 ·Status: ACTIVE_NOT_RECRUITING

Measuring Levels of SMN in Blood Samples of SMA Patients

NCT00061607 ·Status: COMPLETED

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

NCT05839145 ·Status: NOT_YET_RECRUITING ·Phase: NA

Adult Spinal Muscular Atrophy (SMA) China Registry

NCT05618379 ·Status: ACTIVE_NOT_RECRUITING

Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments

NCT05768048 ·Status: RECRUITING

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952 ·Status: COMPLETED ·Phase: PHASE1

A Registered Cohort Study on SMA

NCT04010604 ·Status: RECRUITING

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

NCT05335876 ·Status: RECRUITING ·Phase: PHASE3

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

NCT02550691 ·Status: TERMINATED ·Phase: NA

Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA

NCT05866939 ·Status: UNKNOWN

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients with Gaucher Disease Type I

NCT06162338 ·Status: RECRUITING ·Phase: NA

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

NCT06562283 ·Status: RECRUITING ·Phase: NA

Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy

NCT06641895 ·Status: RECRUITING ·Phase: EARLY_PHASE1

SMN Circular RNAs as Potential New Targets and Biomarkers for SMA

NCT05760209 ·Status: UNKNOWN

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

NCT06818838 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.

NCT07058662 ·Status: RECRUITING ·Phase: PHASE1/PHASE2