LYSOSOMAL STORAGE DISEASE
Clinical trials for LYSOSOMAL STORAGE DISEASE 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: LYSOSOMAL STORAGE DISEASE
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: ⭐️ CURE ⭐️
Last updated Mar 18, 2026 14:41 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: LYSOSOMAL STORAGE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Mar 09, 2026 14:23 UTC
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Scientists seek clues in rare cholesterol disorders
Knowledge-focused CompletedThis study aimed to better understand a group of rare genetic disorders where the body cannot properly make or process cholesterol. Researchers collected blood, urine, tissue samples, and medical information from 342 people with these conditions or their family members. The goal …
Matched conditions: LYSOSOMAL STORAGE DISEASE
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Apr 02, 2026 01:11 UTC