GAUCHER DISEASE, TYPE 1
Clinical trials for GAUCHER DISEASE, TYPE 1 explained in plain language.
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Could a single shot replace daily gaucher meds?
Disease control Recruiting nowThis study tests a one-time gene therapy (LY3884961) for adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or stop the need for current enzyme replacement or substrate reduction therapy. About 15 participants will receive a single intravenous dos…
Matched conditions: GAUCHER DISEASE, TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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One-Time gene shot aims to tame rare enzyme disorder
Disease control Recruiting nowThis early-phase trial tests a single injection of gene therapy (LY-M001) in 12 adults with Gaucher disease type 1, a genetic condition causing enzyme deficiency and organ damage. The therapy delivers a working gene to the liver to produce the missing enzyme. The main goals are s…
Matched conditions: GAUCHER DISEASE, TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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Gene therapy could free gaucher patients from lifelong infusions
Disease control Recruiting nowThis study tests a one-time gene therapy called FLT201 for adults with Gaucher disease type 1. Participants currently on regular enzyme replacement therapy will stop that treatment after receiving FLT201. The goal is to see if the gene therapy can keep blood counts stable and con…
Matched conditions: GAUCHER DISEASE, TYPE 1
Phase: PHASE3 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated May 07, 2026 18:40 UTC
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Can a simple test spot Parkinson's 20 years early? new study aims to find out.
Knowledge-focused Recruiting nowThis study is for people with a change in the GBA1 gene, which raises the risk of Parkinson's disease. Researchers will use 15 simple, non-invasive tests to look for very early signs of Parkinson's that can appear up to 20 years before symptoms start. The goal is to create a mode…
Matched conditions: GAUCHER DISEASE, TYPE 1
Sponsor: Shaare Zedek Medical Center • Aim: Knowledge-focused
Last updated May 14, 2026 12:02 UTC