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Post-authorization safety registry for voretigene neparvovec in biallelic RPE65 mutation-associated retinal dystrophy
jRCT2033230516Recruiting
- Sponsor
- Novartis
- Start
- Aug 2023
- Modality
- Gene Therapies
01
Overview
Open-label, single-arm trial of voretigene neparvovec in RPE65 deficiency, sponsored by Novartis. Started Aug 2023; status: recruiting.
02
Drugs
voretigene neparvovec
03
Conditions
RPE65 deficiency
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