One-Time gene shot could change lives of rett syndrome patients
NCT ID NCT05606614
First seen Nov 21, 2025 · Last updated May 23, 2026 · Updated 24 times
Summary
This study tests a one-time gene therapy called TSHA-102 for females with Rett syndrome, a severe genetic disorder. The therapy is given as a single injection into the spine. The goal is to see if it is safe and can help patients regain lost skills like walking or talking. The study involves 15 participants aged 6 to 22.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Email: •••••@•••••
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CHU St. Justine
RECRUITINGMontreal, Quebec, Canada
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
Contact Email: •••••@•••••
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UC San Diego
RECRUITINGLa Jolla, California, 92093, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UT Southwestern Children's Medical Center
RECRUITINGDallas, Texas, 75930, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Washington University, St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.