Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

NCT03837184 · clinicaltrials.gov ↗

PHASE3

Phase

COMPLETED

Status

2

Enrollment

INDUSTRY

Sponsor class

Conditions

Spinal Muscular Atrophy Type I

Interventions

BIOLOGICAL: Onasemnogene Abeparvovec-xioi

Sponsor

Novartis Gene Therapies

Collaborators

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