News

October 2, 2018

AVROBIO Advances AVR-RD-02 Gene Therapy into Gaucher Disease Phase 1/2 Trial in Canada

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AVROBIO is advancing its gene therapy for Gaucher disease, AVR-RD-02, into a Phase 1/2 clinical trial after Health Canada raised no objections to its clinical trial application, the company announced. The trial ... Read more

September 25, 2018

Lab-grown Bone Cells May Predict Gaucher Patients’ Response to Bone Disease Therapy

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Generating specific bone cells derived from Gaucher patients may be used to monitor the progression of bone disease and predict treatment response in Gaucher disease patients, according to researchers. Their ... Read more

September 21, 2018September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

September 20, 2018

Oral Medication Linked to Better Quality of Life Over Infusion Therapy in Gaucher Disease, Study Suggests

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When it comes to treating Gaucher disease, oral medication is associated with a better quality of life, compared with infusion therapy, an interview-based study suggests. Led by researchers at the ... Read more

September 18, 2018

Liver Fibrosis Common in Gaucher Type 1, and ERT Is Seen to Be Beneficial, Study Reports

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Liver fibrosis is a common condition in patients with Gaucher disease type 1 (GD1) and can be predicted by factors such as disease severity and spleen removal, according to new ... Read more

September 13, 2018

Early Gene Therapy Holds Promise to Treat Gaucher Disease, Mouse Study Shows

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Delivery of a systemic gene therapy restored the production of glucocerebrosidase — the deficient enzyme in Gaucher disease — in several organs, eased symptoms, and prolonged survival in mouse models of Gaucher ... Read more

September 11, 2018

Sanofi’s Cerezyme Should Be Administered Under Current Guidelines with Potential for Change, Study Says

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Newly discovered data on the activity of glucocerebrosidase after treatment with Sanofi Genzyme’s Cerezyme (imiglucerase) — an enzyme replacement therapy for Gaucher disease — supports current guidelines for its administration every two ... Read more

September 6, 2018

Multiparametric MRI May Help Identify, Monitor Bone Lesions in Gaucher Patients, Study Suggests

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Using magnetic resonance imaging (MRI) to assess water- and fat-associated tissue changes may be used to identify and monitor bone damage in young people with Gaucher disease, researchers from Egypt ... Read more

September 4, 2018

Type 3 Gaucher Disease Is Most Common Subtype in Filipinos, Study Shows

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Most Filipino patients with Gaucher disease carry severe mutations in the GBA gene and exhibit Gaucher disease type 3, researchers from the Philippines found. The study, “Genetic and clinical characteristics of ... Read more

August 14, 2018August 14, 2018

Blood Infection Kills Untreated Gaucher Patients at Higher Rate, Study Finds

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Patients with Gaucher disease type 1 who never received treatment with enzyme replacement therapy died of septicemia — a blood infection — nine times more often than the general population, followed by liver disease, ... Read more