Testing a gene therapy for AADC deficiency in young children
Part of Brain & nervous system, Genetic & congenital clinical trials.
This early-stage trial tests VGN-R09b, a gene therapy, given to children with AADC deficiency who are not doing well on standard medicines. It aims to improve symptoms and quality of life by addressing the underlying cause of AADC deficiency.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is at least 2 years old but under 8 years old (or has enough head size for surgery)
- A past diagnosis of AADC deficiency with lab confirmation of the specific brain/chemical pattern or a matching gene change
- Motor progress is very limited at the start (about 3 months or less), and usual medicines didn’t help
- Medicines have been stable for a set time (no new AADC medicines for 6 months, no dose changes for 3 months)
- You, as a parent/guardian, can give consent and agree to follow study visits and assessments
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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