News

The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a marketing authorization for Yargesa (miglustat), for the treatment of adults with mild-to-moderate type 1 Gaucher disease. Yargesa is a generic of Zavesca, which has been authorized in the European…

A multicenter study shows that people with type 1 Gaucher disease (GD) may have higher levels of serum autoantibodies, but these patients showed no clinical signs of autoimmune disorders. The study, “Prevalence of autoantibodies in the course of Gaucher disease type 1: A multicenter study comparing Gaucher disease patients…

Combining oral Zavesca (miglustat) with intravenous enzyme replacement therapy (ERT) may prevent neurological problems in patients with neuronopathic Gaucher disease, a case study suggests. The research, “Combination Therapy In A Patient With Chronic Neuronopathic Gaucher Disease: A Case Report,” was published in the Journal of Medical Case…

The drug compound lucerastat is well-tolerated overall and does not seem to cause any serious adverse side effects according to a study conducted by scientists at Actelion Pharmaceuticals Ltd.

Noninvasive techniques measuring shear wave velocity in body tissues show potential at distinguishing Gaucher disease patients from other diseases with common symptoms, such as enlarged spleen. The study, “Are transient and shear wave elastography useful tools in Gaucher disease?” was published in the journal Blood Cells, Molecules & Diseases. Early…

Enzyme replacement therapy (ERT) in Gaucher disease patients has no significant impact on patients’ final height, suggests a small group retrospective study. The study, “Growth and final height of children with Gaucher disease: A 15-year follow-up at an Israeli Gaucher center,” was published in the journal Blood Cells,…

Enzyme replacement therapy (ERT) with Cerezyme (imiglucerase) in children with Gaucher disease type 3 (GD3) led to marked and quick improvement in key disease manifestations, with benefits lasting for up to five years, a retrospective, worldwide study reported. These and other outcomes seen led the researchers to recommend, based on “clear evidence,”…

Patients with Gaucher disease type 1 (GD1) have a high risk of forming gallstones. Researchers now have connected specific gene variations and other risk factors, such as higher body mass index (BMI) and cholesterol values, to patients with higher occurrence of gallstones. The study “Cholelithiasis in Patients with…

Delaying the start of enzyme replacement therapy (ERT) may lead to worse symptoms and, potentially, permanent damage in patients with Gaucher disease (GD). The study, “Time of initiating enzyme replacement therapy affects immune abnormalities and disease severity in patients with Gaucher disease,” published in PLOS One, underscores the importance of…

Making lysosomes more acidic may be an ingeniously simple, yet efficient, way to prevent the consequences of lysosomal storage disorders such as Gaucher disease. And restoring acidity can be achieved with repurposed drugs that are already in use for other diseases. The discovery by researchers at the University of…