Study design & results
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Voretigene neparvovec in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy
2019-003781-41Phase 3
- Sponsor
- Novartis
- Start
- Nov 2026
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of voretigene neparvovec in RPE65 deficiency, sponsored by Novartis. Primary endpoint: Change from baseline in full-field light sensitivity threshold.
02
Drugs
voretigene neparvovec
03
Conditions
RPE65 deficiency
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