METACHROMATIC LEUKODYSTROPHY
Clinical trials for METACHROMATIC LEUKODYSTROPHY explained in plain language.
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New stem cell approach shows promise for rare childhood diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to see if a donor's stem cells can successfully take over the patient's bone marrow while keeping side effects low. Participants receive chemotherapy…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 26, 2026 08:19 UTC
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Gene therapy hope for kids with rare nerve 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 nervous system. The treatment uses the patient's own blood stem cells, modified to produce a missing enzyme, to slow …
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 26, 2026 08:08 UTC
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New york babies to get extra screening for rare diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study offers parents of newborns in select New York hospitals the option to screen their baby for a panel of rare genetic disorders, beyond standard newborn screening. The goal is to learn how accurate the tests are, how common these conditions are, and whether early diagnos…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated May 26, 2026 08:09 UTC