New gene therapy trial offers hope for kids with rare brain disorder
NCT ID NCT04903288
First seen Oct 31, 2025 · Last updated May 10, 2026 · Updated 27 times
Summary
This study tests a gene therapy called eladocagene exuparvovec in 13 children with AADC deficiency, a rare genetic disorder that affects brain function. The therapy is given directly into the brain using a special needle. The main goals are to check safety and see if it raises levels of a key brain chemical called HVA. The study includes short-term and long-term follow-up to track benefits and side effects.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Chaim Sheba Medical Center
Ramat Gan, 5262000, Israel
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Hospital
Durham, North Carolina, 27705, United States
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National Taiwan University Hospital, Department of Pediatrics and Medical Genetics
Taipei, 10041, Taiwan
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Texas Children's Hospital
Houston, Texas, 77030, United States
Conditions
Explore the condition pages connected to this study.