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: PHASE3 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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One-Time gene shot could free gaucher patients from lifelong infusions
Disease control Recruiting nowThis 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: PHASE1, PHASE2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:37 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: PHASE1, PHASE2 • 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