Gene therapy for MPS IIIA to slow brain decline
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests a gene-transfer treatment (delivered into the spinal fluid) for children with MPS IIIA, a rare genetic condition. It aims to improve or slow down brain and developmental changes caused by a missing enzyme.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Have confirmed MPS IIIA with very low or no SGSH enzyme activity
- Genetic testing must show specific SGSH gene changes (happening in both copies)
- Be within the study age ranges (starting from about 3–6 months up to 5 years, depending on site/cohort)
- Be able to safely get a spinal tap (lumbar puncture) and an MRI
- Must not have certain gene or health test results that could raise risk or affect results
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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