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2 clinical studies listed.
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Tundra lists 2 Biallelic RPE65 Mutation-associated Retinal Dystrophy clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT04516369
Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.
Gender: All
Ages: 4 Years - 100 Years
Updated: 2025-04-25
1 state
NCT05858983
Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
Gender: All
Ages: 8 Years - 45 Years
Updated: 2023-05-15
1 state