Small molecule chaperone therapies may play an increasingly important role in the treatment of lysosomal storage disorders such as Gaucher disease, according to a review published in the journal Neural Regeneration Research.
A new screening device called the Seeker System, which detects lysosomal storage disorders (LSDs) like Gaucher disease in newborns, has received de novo clearance from the U.S. Food and Drug Administration (FDA). The de novo classification offers an alternative pathway to classify novel types of medical devices of…
An analysis of four clinical trials testing Cerdelga (eliglustat) for Gaucher disease patients shows the treatment is safe and without serious adverse events. The study, “A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency,…
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,”…
