Clinical Trial: A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease

U.S., Feb. 12 -- ClinicalTrials.gov registry received information related to the study (NCT06818838) titled 'A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease' on Jan. 24.

Brief Summary: Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic "crumpled tissue paper" appearance, leading to pathological changes in involved ...