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March 28, 2017March 28, 2017

Researchers Review Gaucher Disease Characteristics, Clinical Presentation, Treatments

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A review of the physiological mechanisms, clinical manifestations, and available treatments of Gaucher disease (GD) has been published. The study, ”A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments,” ... Read more

March 23, 2017

New Model Developed for Predicting Long-Term Complications in Type 1 Gaucher’s

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A new model for predicting the risk of long-term complications in patients with type 1 Gaucher disease (GD) was designed based on a 17-year study that measured a naturally-occurring enzyme ... Read more

March 21, 2017

Study in Monkeys Identifies Ambroxol as Possible Therapy for Gaucher Disease

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Researchers suggest that a compound called ambroxol should be explored in clinical trials as a potential drug for Gaucher disease. Their study in non-human primates showed that the drug increased the ... Read more

March 16, 2017March 16, 2017

Identifying the Bone Disorder That a Gaucher Patient Has Is Important, Study Concludes

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Bone disorders in Gaucher disease patients need to be specially considered, suggests a study published in the Journal of Bone and Mineral Research. This can help with early diagnosis and ... Read more

March 14, 2017

Experts Establish New Set of Goals for Managing Gaucher Disease

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Great effort should be made to improve quality of life, reduce fatigue and encourage normal participation in school and work activities in people with Gaucher disease. These are some of ... Read more

March 7, 2017

Enzyme Replacement Therapy in GD May Affect Immune System Performance, Study Suggests

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Enzyme replacement therapy (ERT) was associated with decreased levels of inflammatory mediators in a group of patients with Gaucher disease (GD) type 1. The study “Could enzyme replacement therapy promote ... Read more

March 2, 2017

Suppressing Inflammation-promoting Protein Could Lead to Gaucher Therapies, Study Reports

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Suppressing a protein that promotes the inflammation associated with Gaucher and other lysosomal storage diseases could lead to new treatments for the conditions, researchers say. Scientists from the Cincinnati Children’s ... Read more

February 27, 2017

Lost Function in Gaucher Cells Partially Restored by Coenzyme Q10, Researchers Say

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Coenzyme Q10 (CoQ), a naturally occurring antioxidant, partially restored the function of a cell model of Gaucher disease. These artificially produced cells can be used in the future to study the ... Read more

February 21, 2017

Gaucher Disease Genetic Screening Does More Good Than Harm, Researchers Argue

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Genetic screening for Gaucher disease identifies many patients that may never experience symptoms, according to a new study, and researchers argue that the benefits of early diagnosis of potentially harmful genetic ... Read more

February 16, 2017

Cerdelga Pills as Maintenance for Gaucher Patients as Effective as Injectable Cerezyme, Phase 3 Trial Finds

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Cerdelga (eliglustat) pills are as effective and safe as the injectable drug Cerezyme (imiglucerase) as a maintenance therapy for Gaucher disease after stabilizing patients on Cerezyme, according to the Phase ... Read more