A targeted therapy currently being investigated for treating certain types of cancer can overcome some of the limitations of standard enzyme replacement therapy and be a promising therapeutic avenue for Gaucher disease and other lysosomal storage disorders, a study says. The therapy, which uses small cellular carriers to…
Magnetic resonance spectroscopy (MRS) — a specialized imaging test to visualize and measure the amount of fat in certain regions of the body — may help monitor disease severity and treatment response in people with Gaucher disease, a pilot study suggests. The study, “Gaucher…
The levels of a molecule known as CCL18 in the blood can be used in routine clinical practice to assess disease severity in patients with Gaucher disease, a study reports. The study, “Accuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease…
The U.S. Food and Drug Administration (FDA) has approved Avrobio‘s application to expand to the U.S. its ongoing Phase 1/2 trial testing its gene therapy candidate AVR-RD-02 in people with type 1 Gaucher disease. The trial (NCT04145037), called GAU-201, is investigating the safety and efficacy…
Gaucher disease is among the more costly of chronic diseases to treat in Iran, and efforts are needed to minimize the financial burden facing patients and health authorities in the country, researchers say. Findings of their study, “Healthcare resource utilization and cost of care for Gaucher patients in…
Cerdelga (eliglustat) has a favorable long-term safety profile consistent with previous observations among patients with type 1 Gaucher disease, pooled data from four completed trials show. The findings of the study, “Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher…
Although rare, aneurysms of the splenic artery are most likely underreported among patients with Gaucher disease, a case report study says. The case report, “Splenic Artery Aneurysms, a Rare Complication of Type 1 Gaucher Disease: Report of Five Cases,” was published in the Journal of Clinical Medicine.
The plant-based enzyme replacement therapy Elelyso (taliglucerase alfa) improves bone marrow response in adult patients with type 1 Gaucher disease, according to researchers. The findings of the study, “Improvement in bone marrow infiltration in patients with type I Gaucher disease treated with taliglucerase alfa,” were published in The…