Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
N/A
In this observational study, researchers are looking at the effects of spin...
Status
Recruiting
Location
St. Jude Children's Research Hospital
Start Date
2025-10-29
Trial ID
NCT06532474
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
PHASE3
This trial employs a multicenter, randomized, open-label, standard-of-care-...
Status
Recruiting
Location
The Seventh Medical Center of Chinese PLA General Hospital
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
PHASE4
In this study, researchers will learn more about the use of nusinersen (BII...