Posts by: Alice Melao

News

Enzyme replacement therapy with Cerezyme (imiglucerase) shows long-term benefits on the quality of life in young children with Gaucher disease type 1 (GD1), a study published in Molecular Genetics & Genomic Medicine ... Read more

March 20, 2018March 20, 2018

Late Gaucher Diagnosis Can Worsen Health and Quality of Life, Case Study Shows

News

Late diagnosis and treatment of Gaucher’s disease, particularly when there are bone symptoms, can worsen a patient’s health and quality of life, a case study shows. It involved a Turkish woman ... Read more

March 13, 2018March 13, 2018

Enzyme Replacement Therapy and Bisphosphonates Can Limit Gaucher Bone Loss, Study Shows

News

Enzyme replacement therapy, either alone or combined with a bone loss prevention treatment, helps patients with Gaucher disease type 3 avoid bone thinning, a long-term study shows. The treatment was ... Read more

February 15, 2018February 15, 2018

Possible Treatment for Neuropathic Gaucher Disease, Arimoclomol, Shows Potential in Early Study

News

Arimoclomol, an investigative therapy being developed by Orphazyme, increases glucocerebrosidase activity in cells from Gaucher patients, including those with neuropathic forms of the disease, a new study says. Glucocerebrosidase is ... Read more

February 8, 2018February 8, 2018

MRE Analysis of Liver Stiffness Helps Monitor Disease Severity in GD Type 1 Patients

News

Using non-invasive magnetic resonance elastography (MRE) to evaluate liver stiffness can help monitor disease severity and progression in Gaucher disease type 1 (GD1) patients. That finding was reported in the ... Read more

December 14, 2017

ERT Infusion Sessions Could Be Cut from 1 Hour to 10 Minutes, Gaucher Research Shows

News

Reducing infusion time of Vpriv (velaglucerase alfa) enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease from one hour to 10 minutes may be possible using a time-reducing ... Read more

November 28, 2017

Gaucher Outcome Survey Patient Registry Provides Global Insights into Disease

News

Results from the ongoing Gaucher Outcome Survey (GOS) continue to provide new insights into Gaucher disease (GD) characteristics, as well as improved assessments of treatment patterns and long-term global outcomes. ... Read more

November 21, 2017

Abcertin Clinical Data Supports Use as Alternative Replacement Therapy for Type 1 GD

News

Phase 3 clinical data demonstrated that Abcertin, an investigational formulation of imiglucerase being developed by ISU Abxis, is a safe and effective therapeutic option for the treatment of patients with ... Read more

November 7, 2017

Baby with Gaucher Type 2 Not Inherited from Mother Detailed in Case Study

News

The case of an infant with type 2 Gaucher disease who clearly inherited one disease-causing mutation from his father but whose mother did not show evidence of the second defective ... Read more

October 26, 2017

Why Variations in the Neurological Manifestations of Gaucher Make It Difficult to Diagnose

News

A major challenge of diagnosing Gaucher disease is that its neurological manifestations can appear very early in life or much later, according to a National Institutes of Health review of the rare condition. ... Read more