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Open-label Phase 3 Voretigene Neparvovec Gene Therapy in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
jRCT2080225359Phase 3Completed
- Sponsor
- Novartis
- Start
- Aug 2020
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of voretigene neparvovec in RPE65 deficiency, sponsored by Novartis. Started Aug 2020; status: completed.
02
Drugs
voretigene neparvovec
03
Conditions
RPE65 deficiency
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