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October 10, 2019

Gaucher Disease Might Be Associated with Rare Types of Blood Cancer, Case Report Suggests

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Gaucher disease can predispose patients to rare types of blood cancer, and doctors should be vigilant for the development of these malignancies, a case report suggests. The study, “A ... Read more

October 8, 2019October 17, 2019

Screening for Multiple Myeloma and Other Blood Cell Disorders May Be Useful with Gaucher, Study Says

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Gaucher disease is not common in people with a group of disorders called plasma cell dyscrasias (PCDs), which include multiple myeloma. But screening for these disorders among Gaucher patients could ... Read more

October 7, 2019

New Fly Model of Gaucher Disease May Improve Knowledge of Disease Mechanisms, Researchers Say

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A new fly model of Gaucher disease may more fully summarize and present the molecular complexities of the disease in humans than other animal models do, researchers say. This ... Read more

October 3, 2019

Pilot Study Demonstrates Feasibility of Gaucher Monitoring with Cell Phones

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Mobile phone-based monitoring may allow for better measurement of how Gaucher disease affects the lives of those who have it, a new pilot study reports. The investigators suggest that ... Read more

September 30, 2019

IGA Uses Tattoos, ‘Rare Stars’ to Mark Oct. 1, International Gaucher Day

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What began in the early 1990s as an informal gathering of seven European patient groups in the Italian port of Trieste has evolved into the world’s largest charity for ... Read more

September 26, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 23, 2019

Toddler Becomes Youngest Patient to Develop Gaucheroma While on Enzyme Replacement Therapy, Case Study Reports

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A 15-month-old boy has become the youngest reported patient with Gaucher disease to develop multiple benign accumulations of Gaucher cells — called a Gaucheroma — after starting on enzyme ... Read more

September 19, 2019September 19, 2019

Ambroxol Plus ERT Can Ease Neurological Symptoms in Some Gaucher Type 3 Patients, Case Study Suggests

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Adding ambroxol to enzyme replacement therapy (ERT) lowers the levels of glucosylsphingosine (lyso-Gb1) — a known diagnostic and response biomarker of Gaucher disease — and can alleviate, to some extent, neurologic ... Read more

September 16, 2019

FDA Asked to Allow Higher Dose of PROO1 Gene Therapy in Gaucher Type 2 Clinical Trial

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Prevail Therapeutics is asking the U.S. Food and Drug Administration (FDA) to approved a request to open clinical trials, at higher doses than initially planned, into its investigational gene therapy ... Read more

September 12, 2019

‘Simple’ Algorithm Seen to Help Diagnose Rare Diseases Like Gaucher Type 3

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A new algorithm that examines neurological and oculomotor signs may make it much easier to diagnose certain “often overlooked” rare neurological conditions, including Gaucher disease type 3 (GD3), the research ... Read more

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