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, 12 tagged with Gaucher disease type III itself.
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Cough medicine repurposed for rare brain disease in kids
Disease control CompletedThis study tested high doses of Ambroxol, a common cough medicine, in 12 children aged 3 to 18 with Type 3 Gaucher disease, a rare genetic disorder affecting the brain and body. The goal was to see if it is safe and can improve movement problems like ataxia. The trial took place …
Sponsor: Agyany Pharma LTD • Aim: Disease control
Last updated Aug 26, 2026 00:00 UTC
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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
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Enzyme therapy shows promise for rare gaucher type 3 in small chinese study
Disease control CompletedThis study tested the enzyme replacement drug imiglucerase (Cerezyme) in 12 Chinese patients with Gaucher disease type 3, a rare genetic disorder that affects the brain and body. Participants received the highest approved dose (60 units per kilogram) every two weeks for one year.…
Phase 4 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 26, 2026 17:18 UTC