GAUCHER DISEASE TYPE 1
Clinical trials for GAUCHER DISEASE TYPE 1 explained in plain language.
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Gene therapy trial aims to fix enzyme defect in gaucher disease
Disease control CompletedThis early-stage trial tested a new gene therapy called FLT201 in 10 adults with Gaucher disease type 1. The therapy uses a harmless virus to deliver a working copy of the gene that produces a missing enzyme. The main goal was to check safety and see if the treatment can boost en…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE1 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Could an antioxidant help gaucher disease? new study investigates
Knowledge-focused CompletedThis study measured levels of brain chemicals related to oxidative stress and inflammation in people with type 1 Gaucher disease and healthy volunteers. Researchers gave 33 participants with Gaucher disease the antioxidant N-acetylcysteine (NAC) for about 90 days to see if it cha…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE2 • Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC