Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
NCT03461289
·
clinicaltrials.gov ↗
PHASE3
Phase
COMPLETED
Status
33
Enrollment
INDUSTRY
Sponsor class
Conditions
SMA
Interventions
BIOLOGICAL:
Onasemnogene Abeparvovec-xioi
Sponsor
Novartis Gene Therapies