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
54 clinical trials for this condition and its sub-types.
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Cough medicine repurposed for rare brain disease in kids
Disease control TerminatedThis 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 …
Phase: NA • Sponsor: Agyany Pharma LTD • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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VR headsets tested as a possible treatment for blindness
Disease control TerminatedThis study tested whether using a virtual reality headset for one-hour sessions could help regenerate damaged optic nerves and improve vision in people with glaucoma or other retinal diseases. The idea came from promising results in rodents. However, the trial was terminated earl…
Phase: NA • Sponsor: Stanford University • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC