When given over two decades, Cerezyme (imiglucerase) was effective at easing most symptoms of Gaucher disease type 1 (GD1), according to long-term data from an international patient registry. Importantly, these findings were consistently observed across different patient subgroups, and were not influenced by disease severity, time of therapy…
A single infusion of AVR-RD-02, Avrobio’s experimental gene therapy for Gaucher disease type 1, was able to lower the levels of two disease biomarkers within three months in the first patient dosed in an ongoing Phase 1/2 trial, the company said. The investigational therapy also was safe…
Liver steatosis, a condition characterized by the buildup of fatty tissue inside the liver, is common in adults with type 1 Gaucher disease and is strongly associated with metabolic changes linked to fatty liver disease, an Italian study has found. The condition is also associated with liver scarring…
Centogene and Evotec have expanded their collaboration agreement to focus on the development of new treatments for Gaucher disease and other rare disorders caused by mutations in the GBA gene, the companies announced. This expanded partnership builds upon a previous collaboration agreement, launched in 2018,…
Lysogene has entered into a collaborative research agreement with Yeda Research and Development, a commercial branch of the Weizmann Institute of Science, to develop a gene therapy for neuronopathic Gaucher disease that affects the brain and spinal cord, the biopharmaceutical company announced. The therapy also…
Arimoclomol, Orphazyme’s investigational therapy for Gaucher disease and other disorders, leads to clinically meaningful, dose-dependent reductions in liver and spleen size in patients with type 1 or 3 Gaucher, according to top-line results of a Phase 2 trial. Despite failing at its primary…
Using a non-invasive high-resolution imaging method, physicians found that long-term treatment with Cerezyme (imiglucerase) followed by a switch to Cerdelga (eliglustat) seemed to prevent or delay bone loss in a man with type 1 Gaucher disease. The findings were described in a case report, “…
Although standard therapies for Gaucher disease (GD) usually reduce liver damage, some forms of treatment — particularly enzyme replacement therapy (ERT) — seem to be linked to a higher incidence of fatty liver disease, a study found. The findings highlight the importance of constantly monitoring liver health…
Components of the RANK pathway — a biological pathway that acts as a bridge between the immune and skeletal systems — may be used as markers of bone disease progression in patients with Gaucher disease, a study says. The study, “Effect of Substrate Reduction…
Non-motor symptoms, such as cognitive impairments and depression, may be used as early predictors of parkinsonism in Gaucher disease patients on enzyme replacement therapy (ERT), according to a recent study. The study, “Cognitive decline and depressive symptoms: early non-motor presentations of parkinsonism among Egyptian Gaucher…