Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Clinical trial NCT03837184 · COMPLETED
Trial details
- NCT number
- NCT03837184
- Sponsor
- Novartis Gene Therapies
- Status
- COMPLETED
- Phase
- Phase 3
- Study type
- INTERVENTIONAL
- Start date
- 2019-05-31
- Primary completion
- 2021-06-29
- Enrollment
- 2
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