GAUCHER DISEASE, TYPE 1
Clinical trials for GAUCHER DISEASE, TYPE 1 explained in plain language.
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One-Time gene shot could free gaucher patients from lifelong infusions
Disease control Recruiting nowThis early-stage trial tests a single-dose 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 regular enzyme replacement therapy. About 15 participants will receive one intravenous dose and be foll…
Matched conditions: GAUCHER DISEASE, TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated May 26, 2026 02:18 UTC
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Single gene shot could tame rare enzyme disorder
Disease control Recruiting nowThis study tests a one-time gene therapy injection for adults with Gaucher disease type 1, a rare genetic condition that causes organ enlargement and bone problems. The therapy uses a harmless virus to deliver a working gene to the liver, so the body can produce the missing enzym…
Matched conditions: GAUCHER DISEASE, TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated May 26, 2026 02:10 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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Parkinson's warning signs found decades early in gene carriers
Knowledge-focused Recruiting nowThis study looks at people with a change in the GBA1 gene, which raises their risk for Parkinson's disease. Researchers will use simple, non-invasive tests to find early signs of Parkinson's that can appear up to 20 years before symptoms start. They will compare GBA1 carriers, pe…
Matched conditions: GAUCHER DISEASE, TYPE 1
Sponsor: Shaare Zedek Medical Center • Aim: Knowledge-focused
Last updated May 20, 2026 11:54 UTC