GAUCHER DISEASE TYPE 1
Clinical trials for GAUCHER DISEASE TYPE 1 explained in plain language.
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One-Shot gene therapy trial offers hope for gaucher disease patients
Disease control Recruiting nowThis early-stage study is testing a single intravenous dose of an experimental gene therapy called LY3884961 in adults with Gaucher disease. The main goal is to see if the treatment is safe and tolerable, while also checking if it can improve key disease measures like spleen size…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated Mar 20, 2026 14:48 UTC
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One-Shot gene therapy trial offers new hope for gaucher disease
Disease control Recruiting nowThis early-stage study is testing a new, one-time gene therapy called LY-M001 for adults with Gaucher Disease Type 1. The goal is to see if a single injection is safe and can help the liver produce a crucial missing enzyme for a long time, which could reduce harmful substance bui…
Matched conditions: GAUCHER DISEASE TYPE 1
Phase: PHASE1, PHASE2 • Sponsor: Lingyi Biotech Co., Ltd. • Aim: Disease control
Last updated Mar 18, 2026 14:40 UTC