LYSOSOMAL STORAGE DISEASE
Clinical trials for LYSOSOMAL STORAGE DISEASE explained in plain language.
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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: LYSOSOMAL STORAGE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 26, 2026 08:11 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: LYSOSOMAL STORAGE DISEASE
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 19, 2026 12:00 UTC
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Scientists probe rare cholesterol disorders to unlock secrets
Knowledge-focused CompletedThis study looked at rare genetic disorders where the body cannot make cholesterol properly, which can cause birth defects and learning problems. Researchers collected blood, urine, and tissue samples from 342 participants, including patients and their family members, to better u…
Matched conditions: LYSOSOMAL STORAGE DISEASE
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated May 26, 2026 08:10 UTC