Gaucher disease type 1
Clinical trials for Gaucher disease type 1 explained in plain language.
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One-Time gene shot could free gaucher patients from lifelong infusions
Disease control OngoingThis early-stage study tests a single intravenous dose of a gene therapy called LY3884961 in 15 adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or replace the need for ongoing enzyme replacement or substrate reduction therapy. Participants must…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase 1/2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated Aug 19, 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…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase 2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jul 12, 2026 00:00 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 1/2 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC