- Conditions
- AADC Deficiency
- Interventions
- Eladocagene Exuparvovec
- Genetic
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 4
- States / cities
- Boston, Massachusetts • Durham, North Carolina • Cincinnati, Ohio + 1 more
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- Conditions
- AADC Deficiency
- Interventions
- AAV2-hAADC
- Drug
- Lead sponsor
- Krzysztof Bankiewicz
- Other
- Eligibility
- 24 Months and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2037
- U.S. locations
- 3
- States / cities
- San Francisco, California • Columbus, Ohio