Gene therapy trial hopes to ease rett syndrome in young girls
NCT ID NCT06152237
First seen Oct 31, 2025 · Last updated May 20, 2026 · Updated 23 times
Summary
This study tests a single-dose gene therapy called TSHA-102 in girls aged 5 to 8 with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy is given as a spinal injection and aims to improve symptoms, but it is not a cure. The trial will check for side effects and measure any changes in the girls' condition over up to 6 years.
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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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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.