- Conditions
- Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
- Interventions
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 12 Months and older
- Enrollment
- 87 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Miami, Florida • Iowa City, Iowa + 6 more
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