Gaucher disease
MONDO:0018150Gaucher disease (GD) is a lysosomal storage disorder encompassing three main forms (types 1, 2 and 3), a fetal form and a variant with cardiac involvement (Gaucher disease - ophthalmoplegia - cardiovascular calcification or Gaucher-like disease).
Also known as: Gaucher disease, Gaucher syndrome, acid beta-glucosidase deficiency, glucocerebrosidase deficiency, glucocerebrosidosis, glucosylceramidase deficiency, glucosylceramide beta-glucosidase deficiency, lipoid histiocytosis (kerasin type)
85 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