LYSOSOMAL STORAGE DISEASE
Clinical trials for LYSOSOMAL STORAGE DISEASE explained in plain language.
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Gene therapy shows promise for rare fatal 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 and severe brain disease. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme, and aims to slow or stop disease progres…
Matched conditions: LYSOSOMAL STORAGE DISEASE
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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Gene therapy offers hope for children with rare brain disease
Disease control CompletedThis study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare, inherited brain disease that causes severe disability and early death. The treatment uses the child's own blood stem cells, which are modified in a lab to carry a working copy of the mi…
Matched conditions: LYSOSOMAL STORAGE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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Scientists dive into rare cholesterol disorders to uncover clues
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 families to learn more about these conditions. The g…
Matched conditions: LYSOSOMAL STORAGE DISEASE
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC