Metachromatic leukodystrophy (MLD)
Clinical trials for Metachromatic leukodystrophy (MLD) explained in plain language.
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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…
Matched conditions: METACHROMATIC LEUKODYSTROPHY (MLD)
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…
Matched conditions: METACHROMATIC LEUKODYSTROPHY (MLD)
Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC