Gene therapy study for children with Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study tests a new gene therapy for children aged 7 to 17 with Fabry disease. The treatment aims to fix the faulty gene that causes the condition, which may help reduce symptoms and prevent organ damage.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 7 and 17 years old.
- You must have a confirmed Fabry disease diagnosis through genetic testing.
- You must have at least one symptom of Fabry disease, like pain, rash, or kidney issues.
- Your kidney function needs to be at a certain level.
- You cannot have certain infections like hepatitis, HIV, or syphilis.
- You must not have had other gene therapy or experimental drugs recently.
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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