GAUCHER DISEASE
Clinical trials for GAUCHER DISEASE explained in plain language.
Never miss a new study
Get alerted when new GAUCHER DISEASE trials appear
Sign up with your email to follow new studies for GAUCHER DISEASE, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
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
Phase: EARLY_PHASE1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
-
New york program offers extra screening for 100,000 newborns
Diagnosis ENROLLING_BY_INVITATIONScreenPlus 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 Jun 26, 2026 16:15 UTC
-
NIH launches major study to unravel mysteries of rare genetic 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. 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 Jun 27, 2026 13:06 UTC
-
Home infusions may help patients stick to treatment
Knowledge-focused OngoingThis study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…
Matched conditions: GAUCHER DISEASE
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC