Metachromatic leukodystrophy
MONDO:0018868A rare lysosomal storage disorder characterized by intralysosomal accumulation of sulfatides in various tissues, leading to progressive deterioration of motor and neurocognitive function.
Also known as: MLD, arylsulfatase A deficiency
26 clinical trials for this condition and its sub-types, 20 tagged with Metachromatic leukodystrophy itself.
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Sub-types of Metachromatic leukodystrophy
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Metachromatic leukodystrophy, juvenile form 2 trials · 4 incl. sub-types
2 sub-types
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Can Gene-Modified stem cells stop a devastating brain disease?
Cure Not yet recruitingThis trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare inherited disease that damages the nervous system. Researchers take a patient's own blood stem cells, add a corrected gene, and infuse them back. The goal is to see if this approach is safe and can slo…
Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Cure
Last updated Sep 03, 2026 00:00 UTC
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Gene therapy aims to halt fatal brain disease in children
Disease control Not yet recruitingThis trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…
Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC