Gene therapy study for Tay-Sachs and Sandhoff disease
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study explores whether a gene therapy approach for GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) can work by testing cells from your blood. It aims to understand how this treatment might help your body.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of GM2 gangliosidosis (Tay-Sachs or Sandhoff disease).
- You are at least 5 years old.
- You already have a blood test scheduled as part of your care.
- No one (you or your guardian) has objected to being in the study.
- If you are under 18, you weigh at least 25 kg (about 55 pounds).
- You are not under guardianship, curatorship, or receiving state medical aid (AME).
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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