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 a rare, severe brain disease called metachromatic leukodystrophy (MLD). Five children aged 6 months to 5 years received injections of a harmless virus carrying a working gene directly into their brains. The goal was to see if it …
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 17, 2026 06:19 UTC
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Gene therapy shows promise for rare, devastating brain disease in kids
Disease control CompletedThis study tested a gene therapy called OTL-200 in 10 children with early-onset metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme, and aims to slo…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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Gene therapy offers hope for children with rare 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. The treatment involves taking the child's own blood stem cells, adding a working copy of the faulty gene, and giving the cell…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:55 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 slow nerve damage and improve function. The child received …
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated May 15, 2026 11:53 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 17, 2026 06:23 UTC