Gene therapy trial offers hope for girls with rare brain disorder
NCT ID NCT06152237
First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 21 times
Summary
This study tests a new gene therapy called TSHA-102 for girls aged 5 to 8 with Rett syndrome, a serious genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid to try to improve symptoms. The main goal is to check safety, but researchers will also look for signs of improvement. The study is small (6 participants) and lasts up to 6 years, with close monitoring for side effects.
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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.
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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CHU Ste-Justine
Montreal, Quebec, H3T 1C5, Canada
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Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust
London, United Kingdom
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Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101, United States
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Rush University Medical Center & Children's Hospital
Chicago, Illinois, 60612, United States
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Washington University, St. Louis
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.