New gene therapy hopes to restore milestones in rett syndrome
NCT ID NCT05606614
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a one-time gene therapy called TSHA-102 in females aged 6 to under 22 with classic Rett syndrome. The goal is to see if it is safe and can help them regain or gain new developmental skills like walking or talking. Participants receive the therapy through a spinal injection and are monitored for side effects and improvements.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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