METACHROMATIC LEUKODYSTROPHY
Clinical trials for METACHROMATIC LEUKODYSTROPHY explained in plain language.
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Brain gene injection shows promise for rare childhood disease
Disease control CompletedThis study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare and severe brain disease. Five children aged 6 months to 5 years received a single injection of a harmless virus carrying a healthy gene directly into their brains. The goal was to see i…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Disease control
Last updated May 26, 2026 12:44 UTC
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Stem cell gene therapy offers hope for children with fatal brain disease
Disease control CompletedThis study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare and fatal genetic disease that damages the brain and nerves. Doctors took stem cells from each child, added a working copy of the faulty gene, and put the cells back after mild chemother…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 26, 2026 12:06 UTC
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Experimental enzyme therapy tested in rare child brain disease after transplant
Disease control CompletedThis study tested an experimental enzyme therapy called METAZYM 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 substance buildup…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated May 26, 2026 12:01 UTC
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Gene therapy shows promise for rare, devastating childhood disease
Disease control CompletedThis study tested a one-time gene therapy (OTL-200) in 10 children with early-onset metachromatic leukodystrophy (MLD), a severe genetic disorder that damages the nervous system. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme. The main g…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 19, 2026 12:00 UTC
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Gene sequencing breakthrough may unlock mysterious brain diseases in kids
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete scan of a person's DNA) can help doctors diagnose leukodystrophies—rare brain disorders that damage white matter—more accurately than standard methods. About 236 children with abnormal brain MRI scans but no known g…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 18, 2026 12:01 UTC