GAUCHER DISEASE TYPE 1
Clinical trials for GAUCHER DISEASE TYPE 1 explained in plain language.
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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…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 13:08 UTC
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Gene therapy for gaucher disease: 5-year safety check begins
Disease control OngoingThis study follows 75 people with Gaucher disease type 1 who previously received FLT201 gene therapy. Researchers will monitor them for 5 years to see if the treatment remains safe and if its effects last. The goal is to understand whether a single dose can provide long-term dise…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC