METACHROMATIC LEUKODYSTROPHY
Clinical trials for METACHROMATIC LEUKODYSTROPHY explained in plain language.
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One-Time gene therapy aims to halt devastating childhood brain disease
Disease control OngoingThis study is testing a single infusion of an experimental gene therapy called OTL-200 for children and teenagers with a rare, progressive brain disease called Late Juvenile Metachromatic Leukodystrophy (MLD). The therapy involves collecting a patient's own blood stem cells, addi…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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New hope for kids with devastating genetic diseases
Disease control OngoingThis study is testing a stem cell transplant procedure for children and adults with severe, inherited metabolic disorders and a rare bone disease. The goal is to see if using a specific drug combination can help the donor cells successfully take hold in the patient's body while k…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Mar 13, 2026 15:05 UTC
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Massive study screens 100,000 babies for hidden rare diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to gather information by offering expanded newborn screening to 100,000 babies in New York. It will test for 14 rare genetic disorders to see how accurate the tests are, how often these diseases occur, and if finding them early helps children's health. The main go…
Matched conditions: METACHROMATIC LEUKODYSTROPHY
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC