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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Could a daily pill replace IV infusions for gaucher type 3?
Disease control OngoingThis phase 3 trial tests venglustat, a daily oral drug, against the standard IV treatment Cerezyme in 43 people with Gaucher disease type 3. Participants must have been on enzyme replacement therapy for at least 3 years. The goal is to see if venglustat can stabilize or improve n…
Phase 3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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Could a pill replace infusions for gaucher brain symptoms?
Disease control OngoingThis phase 2 trial tests an oral drug called venglustat, given alongside the standard enzyme therapy Cerezyme, in adults with Gaucher disease type 3 (a rare genetic disorder affecting the body and brain). The study has four parts: first, it checks spinal fluid biomarkers to disti…
Phase 2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC