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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 ... 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 ... 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 ... 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 ... 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 ... 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 ... 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 ... Read more

August 9, 2018

Elelyso Clears Disease Cells from Bone Marrow of GD Type 1, Study Reports

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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 ... Read more

August 7, 2018

More Rapid Infusions of VPRIV Still Safe, Effective for Gaucher Type 1 Patients, Study Finds

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Shortening the duration of VPRIV (velaglucerase alfa) infusions from 60 to 10 minutes does not affect the safety or effectiveness of the enzyme replacement therapy in patients with Gaucher disease type 1, ... Read more

August 2, 2018August 3, 2018

Prenatal Gene Therapy May Prevent Brain Damage in Gaucher Type 2, Mouse Study Suggests

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Prenatal delivery of a gene therapy restored the production of glucocerebrosidase — the deficient enzyme in Gaucher disease — prevented nerve cell death, and prolonged survival in a mouse ... Read more

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