GAUCHER DISEASE
Clinical trials for GAUCHER DISEASE explained in plain language.
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Gene therapy hope for kids with rare gaucher disease
Disease control OngoingThis early-stage study tests a single injection of LY-M001 gene therapy in 9 children aged 6 to 17 with type 1 Gaucher disease. The goal is to see if it is safe and can help the body produce the missing enzyme. Researchers will monitor side effects and measure changes in enzyme l…
Matched conditions: GAUCHER DISEASE
Phase: EARLY_PHASE1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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Scientists hunt for genetic clues in rare storage diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to find genetic, biochemical, and clinical factors that affect how severe Gaucher disease and other lysosomal storage disorders become. Up to 1,000 participants, including patients, carriers, and healthy volunteers, will be evaluated yearly. The goal is to better …
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 17, 2026 05:32 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: GAUCHER DISEASE
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated May 13, 2026 16:03 UTC