METACHROMATIC LEUKODYSTROPHY
Clinical trials for METACHROMATIC LEUKODYSTROPHY explained in plain language.
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One-Time gene infusion offers hope for rare brain disease
Disease control OngoingThis study tests a one-time gene therapy called OTL-200 in 6 people with late juvenile metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The treatment uses the patient's own blood stem cells, modified to produce a missing enzyme, and ai…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New stem cell approach aims to tame rare genetic diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get donor cells to grow in the patient's body while keeping transplant-related deaths low. Participants receive chemotherapy drugs (busulfan and f…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 12, 2026 13:39 UTC
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New study offers extra screening for rare diseases in newborns
Knowledge-focused ENROLLING_BY_INVITATIONThis study gives parents the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. Researchers will test 100,000 babies born in New York hospitals to see how accurate the screening is and how often these conditions…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated May 13, 2026 16:03 UTC