GAUCHER DISEASE
Clinical trials for GAUCHER DISEASE explained in plain language.
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Gene therapy injection aims to tame rare enzyme disorder in kids
Disease control OngoingThis early-stage study tests a single intravenous dose of LY-M001 in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if the treatment is safe and can restore the missing enzyme, potentially reducing the need for lifelong enzyme replacement therapy. Resea…
Matched conditions: GAUCHER DISEASE
Phase: EARLY_PHASE1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated May 26, 2026 06:59 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: GAUCHER DISEASE
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated May 26, 2026 07:03 UTC
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Gene hunt: unlocking the secrets of rare diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to identify genetic, biochemical, and clinical factors linked to disease severity in people with Gaucher disease and other lysosomal storage disorders. Up to 1,000 participants, including patients, carriers, and healthy volunteers, will be evaluated annually. The …
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 22, 2026 13:51 UTC