Gene therapy for rare childhood brain disorder MPS IIIB
Part of Genetic & congenital clinical trials.
This early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child has been diagnosed with MPS IIIB (a confirmed genetic test result)
- Your child is under 6 years old
- Your child can complete a cognitive (thinking skills) assessment and score at least 55
- Your child has not received other experimental treatments for MPS IIIB in the past 4 months
- Your child has never had gene therapy or stem cell transplant treatment
- Your child does not have uncontrolled seizures or a bleeding disorder
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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