GAUCHER DISEASE TYPE 1
Clinical trials for GAUCHER DISEASE TYPE 1 explained in plain language.
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Gene therapy trial tests new approach for rare genetic disorder
Disease control CompletedThis first-in-human study tested the safety and early effects of a new gene therapy called FLT201 in adults with Gaucher disease type 1. The therapy aims to boost a missing enzyme to help the body break down harmful substances that build up in cells. Six adults who were already o…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC
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Could a common supplement help a rare disease? brain scans hold clues
Knowledge-focused CompletedThis study aimed to understand if harmful stress and inflammation in the body and brain are part of Type 1 Gaucher disease. Researchers measured specific chemicals in the blood and brain of 33 adults with the disease and healthy volunteers. They also tested if taking an over-the-…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE2 • Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Mar 17, 2026 13:09 UTC