News

October 19, 2017October 19, 2017

New Method of Diagnosing and Monitoring Gaucher Disease Uses Dried Blood Samples

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Using dried blood samples for measuring the levels of Gaucher disease biomarker glucosylsphingosine is now possible with a newly developed method by a team of researchers at Cincinnati Children’s Hospital ... Read more

October 19, 2017

#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

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As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to ... Read more

October 12, 2017October 12, 2017

Parkinson’s Disease Risk Should Be Discussed Before Gaucher Genetic Testing, Patients Say

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A recent survey of people who were tested to see if they carry Gaucher disease-causing mutations in the GBA gene asked if they believe patients should also be informed about the ... Read more

October 3, 2017

Neuropathic Gaucher Disease Mutations Speed Cognitive Decline in Parkinson’s, Study Finds

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Genetic mutations causing neuropathic Gaucher disease (GD) aggravate cognitive dysfunction in Parkinson’s disease (PD), new research shows. The study “Specifically neuropathic Gaucher’s mutations accelerate cognitive decline in Parkinson’s” was published ... Read more

September 26, 2017

Researchers Suggest Lyso-Gb1 as Gaucher Treatment Response Marker

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Lyso-Gb1 (glucosylsphingosine) may be a sensitive marker of disease burden and treatment response in patients with Gaucher disease type 1, argue researchers from the biotechnology company Shire and Zedek Medical Center ... Read more

September 21, 2017

High Levels of Iron During Therapy Linked to Residual Gaucher Disease, Study Finds

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Persistent high levels of iron in patients with Gaucher disease who are receiving maintenance therapy are associated with residual Gaucher disease. This finding was reported in the study “Iron storage ... Read more

September 19, 2017

Gaucher Disease Guidance Could Help Non-specialists Diagnosis It Better, Study Reports

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Doctors who are not Gaucher disease experts should have straightforward and easy-to-follow diagnosis guidance so patients can be treated earlier, according to a study reporting that it can take years ... Read more

September 7, 2017September 7, 2017

Nerve Cell Damage Causes Pain in Gaucher Disease Type 1 Patients, Italian Study Suggests

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Pain is a common symptom among patients with Gaucher disease (GD). Italian researchers say it’s caused by peripheral nerve cell damage and not only bone damage, as was previously thought. Their ... Read more

September 5, 2017

Three Newly Identify Compounds Show Promise for Types 2 and 3 Gaucher Disease

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Researchers have identified three new compounds that can cross the blood-brain barrier and may represent a new therapeutic avenue for types 2 and 3 Gaucher disease — two forms of ... Read more

August 31, 2017

Accordant’s Gaucher Program Receives NCQA Disease Management Accreditation

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Accordant’s health programs, including its Gaucher disease program, have received a special Disease Management (DM) Accreditation from the National Committee for Quality Assurance (NCQA). NCQA is a non-profit organization that ... Read more