METACHROMATIC LEUKODYSTROPHY
Clinical trials for METACHROMATIC LEUKODYSTROPHY explained in plain language.
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One-Time treatment aims to halt devastating childhood brain disease
⭐️ CURE ⭐️ CompletedThis study tested a one-time gene therapy called OTL-200 for children with early-onset metachromatic leukodystrophy (MLD), a rare and fatal genetic brain disease. The treatment involved collecting a child's own blood stem cells, fixing the genetic error in a lab, and returning th…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: ⭐️ CURE ⭐️
Last updated Mar 18, 2026 14:41 UTC
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Experimental enzyme therapy tested for devastating childhood brain disease
Disease control CompletedThis study tested an experimental enzyme replacement therapy called METAZYM in a single child with a severe, rare brain disease called late infantile MLD. The child had already received a stem cell transplant. Researchers gave intravenous enzyme infusions every other week for 18 …
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
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Doctors test gene fix for devastating childhood brain disease
Disease control CompletedThis early-stage trial tested a gene therapy for metachromatic leukodystrophy (MLD), a rare and severe genetic disorder that damages the brain and nervous system. Researchers collected blood stem cells from 20 pre-symptomatic or early-symptomatic children with MLD, used a modifie…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Mar 09, 2026 14:23 UTC
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Doctors inject gene therapy directly into Children's brains to fight rare fatal disease
Disease control CompletedThis early-stage study tested a new gene therapy approach for children with a rare, severe brain disease called metachromatic leukodystrophy (MLD). Doctors injected a modified virus carrying a healthy gene directly into the brains of five young children (ages 6 months to 4 years)…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Disease control
Last updated Mar 02, 2026 15:21 UTC
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New gene test cuts diagnosis time for devastating childhood brain diseases
Diagnosis CompletedThis study tested whether using whole genome sequencing (a complete genetic map) as a first test could provide faster and more accurate diagnoses for children with rare, inherited brain disorders called leukodystrophies. Researchers compared the new approach to the standard step-…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Diagnosis
Last updated Apr 03, 2026 14:43 UTC