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Gaucher disease
Clinical trials for Gaucher disease explained in plain language.
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One-Time gene shot could free gaucher patients from lifelong infusions
Disease control OngoingThis early-stage study tests a single intravenous dose of a gene therapy called LY3884961 in 15 adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or replace the need for ongoing enzyme replacement or substrate reduction therapy. Participants must…
Matched conditions: GAUCHER DISEASE
Phase 1/2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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Gene therapy shot aims to fix gaucher disease in kids
Disease control OngoingThis early-phase trial tests a single intravenous dose of LY-M001 gene therapy in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if it is safe and can improve key symptoms like liver size and blood markers. Researchers will monitor participants for side…
Matched conditions: GAUCHER DISEASE
Early phase 1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
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New york program offers extra screening for 100,000 newborns
Diagnosis By invitation onlyScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…
Matched conditions: GAUCHER DISEASE
Sponsor: Albert Einstein College of Medicine • Aim: Diagnosis
Last updated Jul 30, 2026 00:00 UTC
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NIH launches major study to unravel mysteries of rare genetic diseases
Knowledge-focused By invitation onlyThis study aims to identify genetic, biochemical, and clinical factors linked to disease severity in people with Gaucher disease and other lysosomal storage disorders. Researchers will evaluate up to 1,000 participants, including patients, carriers, and healthy controls, to bette…
Matched conditions: GAUCHER DISEASE
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC