Gaucher disease type 1
Clinical trials for Gaucher disease type 1 explained in plain language.
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Gene therapy may free gaucher patients from lifelong infusions
Disease control Recruiting nowThis Phase 3 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if a single dose can keep blood counts stable so patients can stop their regular enzyme replacement or substrate reduction therapy. The study will enroll 45 people who…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase 3 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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One-Time gene therapy could change gaucher treatment forever
Disease control Recruiting nowThis study tests a new gene therapy called LY-M001 for adults with Gaucher disease type 1, a genetic disorder that causes organ damage and other health problems. The therapy uses a harmless virus to deliver a working copy of the GBA1 gene to liver cells, aiming to restore the mis…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase 1/2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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Can we predict Parkinson's decades early? new study recruits 600 participants
Knowledge-focused Recruiting nowThis study aims to find early signs of Parkinson's disease in people who carry a change in the GBA1 gene, which puts them at higher risk. Researchers will use simple, non-invasive tests to check for subtle changes in movement, thinking, sleep, and other functions that can appear …
Matched conditions: GAUCHER DISEASE TYPE 1
Sponsor: Shaare Zedek Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:25 UTC
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Cough medicine repurposed: ambroxol registry launches for rare brain diseases
Knowledge-focused Recruiting nowThis study creates a registry to collect real-world information on the safety and effectiveness of ambroxol, a common cough medicine, when used at higher doses for Gaucher disease or GBA-related Parkinson disease. Researchers aim to gather data from 300 patients worldwide who are…
Matched conditions: GAUCHER DISEASE TYPE 1
Sponsor: Shaare Zedek Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 UTC