Gene therapy trial for girls ages 2-4 with Rett syndrome
Part of Brain & nervous system, Genetic & congenital, Mental health clinical trials.
This trial tests a new gene therapy called TSHA-102 for girls with classic Rett syndrome caused by a specific MECP2 gene mutation. It aims to see if the treatment is safe and can help improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a girl between 2 and almost 4 years old.
- You have been diagnosed with classic (typical) Rett syndrome.
- You have a confirmed MECP2 gene mutation that stops the gene from working.
- You and your family must be willing to live near the study site during the first 3 months after treatment.
- You must be willing to receive blood or blood products if needed for side effects.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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