Gene therapy hope for toddlers with rare brain disorder
NCT ID NCT07480564
First seen Mar 21, 2026 · Last updated May 11, 2026 · Updated 8 times
Summary
This study tests a single dose of a gene therapy called TSHA-102 in 3 girls aged 2 to 4 with Rett syndrome, a severe genetic disorder. The therapy is given as a spinal injection and aims to improve symptoms by delivering a working copy of the faulty gene. The main goal is to check safety and see if it helps control the disease.
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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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