LYSOSOMAL STORAGE DISEASE
Clinical trials for LYSOSOMAL STORAGE DISEASE explained in plain language.
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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: LYSOSOMAL STORAGE DISEASE
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: LYSOSOMAL STORAGE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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Rare cholesterol disorder study seeks answers
Knowledge-focused CompletedThis study looks at rare genetic disorders where the body can't make cholesterol properly, which can cause birth defects and learning problems. Researchers collect blood, urine, and tissue samples from affected people and their family members to learn more about these conditions.…
Matched conditions: LYSOSOMAL STORAGE DISEASE
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated May 11, 2026 20:48 UTC