One-Shot gene therapy trial offers hope for devastating neurological disorder
NCT ID NCT05606614
Summary
This study is testing a single injection of an experimental gene therapy called TSHA-102 for females with Rett syndrome. The goal is to see if the treatment is safe and can help improve developmental skills by targeting the genetic root cause of the disease. The study will follow participants for five years after receiving the treatment.
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
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