Gene therapy for MPS IIIB in young children
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests an experimental treatment (tralesinidase alfa) delivered directly into the brain for children with MPS IIIB, a rare genetic disorder that affects thinking and development. The goal is to slow or stop cognitive decline by replacing a missing enzyme.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child has been diagnosed with MPS IIIB confirmed by enzyme testing
- Your child has the severe (non-attenuated) form of MPS IIIB, confirmed by a review committee
- Your child is between 1 and 5 years old with a developmental score below 70 on standard testing
- Your child has not received stem cell therapy, gene therapy, or enzyme replacement therapy for MPS IIIB before
- Your child does not have another brain illness (such as a head injury, meningitis, or brain bleed) that might have caused cognitive problems
- Your child does not have a known MPS IIIB genetic variant or family history linked to the milder (attenuated) form
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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