Metachromatic leukodystrophy, late infantile form
MONDO:0017729Also known as: MLD, late infantile form, arylsulfatase A deficiency, late infantile form
8 clinical trials for this condition and its sub-types, 2 tagged with Metachromatic leukodystrophy, late infantile form itself.
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Experimental enzyme therapy tested in rare brain disease after transplant
Disease control CompletedThis study tested an experimental drug called METAZYM (a lab-made enzyme) in one child with late infantile metachromatic leukodystrophy (MLD) who had already received a stem cell transplant. The goal was to see if the enzyme could improve nerve function and reduce harmful buildup…
Phase 2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New MRI study maps rare brain disease in children
Knowledge-focused CompletedThis study used powerful MRI scans to track how a rare genetic disease called metachromatic leukodystrophy (MLD) damages the brain's white matter in young children. Researchers studied 29 children aged 1 to 6 years with MLD, along with a control group, to better understand how th…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC