New gene therapy hopes to help girls with rare brain disorder

NCT ID NCT05606614

Recruiting now Disease control Sponsor: Taysha Gene Therapies, Inc. Source: ClinicalTrials.gov ↗

First seen Nov 21, 2025 · Last updated May 16, 2026 · Updated 21 times

Summary

This study tests a one-time gene therapy called TSHA-102 for females with Rett syndrome, a rare genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid. The goal is to see if it is safe and can help improve skills like walking, talking, or hand use. The study includes girls and women aged 6 to under 22 years.

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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

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02115, United States

Contact Email: •••••@•••••
CHU St. Justine
RECRUITING

Montreal, Quebec, Canada
Rush University Medical Center
RECRUITING

Chicago, Illinois, 60612, United States

Contact Email: •••••@•••••
UC San Diego
RECRUITING

La Jolla, California, 92093, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
UT Southwestern Children's Medical Center
RECRUITING

Dallas, Texas, 75930, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••
Washington University, St. Louis
RECRUITING

St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.

RETT SYNDROME