Gene Therapy for Infants With Type 2 Gaucher Disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy (VGN-R08b) for infants with Type 2 Gaucher disease, a rare genetic condition that affects the brain and body. The goal is 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
- Your child must be 24 months or younger.
- Gaucher disease must be confirmed by enzyme and genetic tests.
- Your child must have neurological symptoms, like trouble with movement or development.
- Your child must not be able to walk independently (not yet walking on their own).
- No prior gene therapy or cell therapy treatments.
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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