Indication
Biallelic RPE65 Mutation-associated Retinal Dystrophy
2 clinical trials
2 products
Clinical trial
An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal DystrophyStatus: Active (not recruiting), Estimated PCD: 2022-04-05
Product
Voretigene neparvovecClinical trial
A Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal DystrophyStatus: Recruiting, Estimated PCD: 2025-11-30
Product
FT-001