Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

NCT05089656 · clinicaltrials.gov ↗

PHASE3

Phase

COMPLETED

Status

126

Enrollment

INDUSTRY

Sponsor class

Conditions

Type 2 Spinal Muscular Atrophy

Interventions

GENETIC: OAV101
PROCEDURE: Sham control

Sponsor

Novartis Pharmaceuticals