Gaucher disease type I
MONDO:0009265Gaucher disease type 1 is the chronic non-neurological form of Gaucher disease (GD) characterized by organomegaly, bone involvement and cytopenia.
Also known as: Gaucher disease type I, Gaucher disease, noncerebral juvenile, Gaucher's disease type I, Gba deficiency, acid Beta-glucosidase deficiency, non-cerebral juvenile Gaucher disease, Gaucher disease type 1, Gaucher disease, type 1
21 clinical trials for this condition and its sub-types, 12 tagged with Gaucher disease type I itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →-
New oral drug shows promise for kids with gaucher disease
Disease control CompletedThis study tested the safety and effectiveness of eliglustat, an oral medication, in 57 children aged 2 to 17 with Gaucher disease types 1 and 3. Some children also received the standard enzyme therapy imiglucerase. Researchers measured how the drug moves through the body, side e…
Phase 3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
-
Gene therapy trial aims to fix enzyme defect in gaucher disease
Disease control CompletedThis early-stage trial tested a new gene therapy called FLT201 in 10 adults with Gaucher disease type 1. The therapy uses a harmless virus to deliver a working copy of the gene that produces a missing enzyme. The main goal was to check safety and see if the treatment can boost en…
Phase 1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
-
Could an antioxidant help gaucher disease? new study investigates
Knowledge-focused CompletedThis study measured levels of brain chemicals related to oxidative stress and inflammation in people with type 1 Gaucher disease and healthy volunteers. Researchers gave 33 participants with Gaucher disease the antioxidant N-acetylcysteine (NAC) for about 90 days to see if it cha…
Phase 2 • Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC