Gene therapy injection for metachromatic leukodystrophy (MLD)
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a direct gene-therapy injection into the body to treat MLD, a genetic brain condition. It may help replace a faulty gene so the disease process slows or changes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be at least 1 month old and have MLD
- Have genetic testing showing an ARSA gene change that matches MLD
- Have MRI brain findings that fit the MLD scoring system
- Have a parent/guardian (or you, if applicable) sign study consent
- Be able to have an MRI scan during the study
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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