Gaucher disease type III
MONDO:0009267Gaucher disease type 3 is the subacute neurological form of Gaucher disease (GD) characterized by progressive encephalopathy and associated with the systemic manifestations (organomegaly, bone involvement, cytopenia) of GD type 1.
Also known as: Gaucher disease type III, Gaucher disease, Subacute neuronopathic type, Gaucher disease, chronic neuronopathic type, Gaucher disease, juvenile and adult, cerebral, Gaucher disease, subacute neuronopathic type, Gaucher's disease type III, cerebral juvenile and adult form of Gaucher disease, chronic neuronopathic Gaucher disease
12 clinical trials for this condition and its sub-types.
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New pill aims to tackle rare genetic disorder affecting brain and body
Disease control Not yet recruitingThis study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable…
Phase: PHASE2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC
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Gene therapy injection aims to control rare brain disease in children
Disease control Not yet recruitingThis early-stage trial tests a new gene therapy called VGN-R08b for children with type III Gaucher disease, a rare genetic disorder affecting the brain and body. The treatment is given as a single injection into the brain's fluid spaces. The study will monitor safety and measure …
Phase: PHASE1, PHASE2 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC