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...
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
PHASE1
The primary objective of this study is to examine the safety and tolerabili...
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)
PHASE3
In this study, researchers will learn more about the use of nusinersen (BII...