GAUCHER DISEASE
Clinical trials for GAUCHER DISEASE explained in plain language.
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One-Shot gene therapy tested in kids with rare genetic disorder
Disease control OngoingThis early-stage study is testing a single intravenous infusion of an experimental gene therapy called LY-M001 in children and adolescents with Gaucher disease type 1. The main goal is to check if the treatment is safe and tolerable. Researchers will also look for early signs tha…
Matched conditions: GAUCHER DISEASE
Phase: EARLY_PHASE1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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Massive study tracks 1,000 patients to unlock secrets of rare genetic diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to better understand Gaucher disease and related lysosomal storage disorders by observing how symptoms and genetics vary among 1,000 patients over many years. Researchers will track participants annually to identify factors linked to disease severity and explore t…
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:33 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: GAUCHER DISEASE
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC