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March 5, 2020March 5, 2020

Israeli Gaucher Clinic Treats Arabs With Disease Prevalent Among Ashkenazi Jews

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If it weren’t for Gaucher disease, Rania Qwaider of Ramallah, Suheir Sandouka of Beit Hanina, and Rabah Mustafa of Nablus might never have become best friends. All three women ... Read more

March 2, 2020March 2, 2020

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

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Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, ... Read more

February 27, 2020February 27, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

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Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. ... Read more

February 24, 2020February 24, 2020

Blood Levels of CCL18 Can Assess Gaucher Severity in Clinical Practice, Study Suggests

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The levels of a molecule known as CCL18 in the blood can be used in routine clinical practice to assess disease severity in patients with Gaucher disease, a study ... Read more

February 21, 2020March 3, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

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An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 20, 2020February 20, 2020

Cough Medicine Ambroxol Relieves Chronic Bone Pain in Woman With Gaucher, Report Says

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The cough medicine ambroxol may be effective for relieving persistent bone pain in people with Gaucher disease, a case report suggests. The report describes how a high dose of ambroxol ... Read more

February 17, 2020February 17, 2020

Fat ‘Floaters’ Impairing Vision in GD3 Patient Highlight Need for Monitoring, New Therapies, Case Study Reports

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A young man with Gaucher disease type 3 (GD3) developed fat deposits in the eye, called floaters, that impaired his vision despite long-term treatment with enzyme replacement therapy (ERT) ... Read more

February 13, 2020February 13, 2020

FDA Grants Orphan Drug, Rare Pediatric Disease Designations to PR001 Investigational Gene Therapy

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Prevail Therapeutics‘ investigational therapy PR001 for the treatment of Gaucher disease. In addition, the regulatory agency ... Read more

February 12, 2020February 12, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 10, 2020February 10, 2020

Thermo Fisher Develops Test to Screen Genes Linked to Gaucher, Other Hereditary Disorders

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Thermo Fisher Scientific said it has developed a new test that will allow reproductive health investigators to screen 420 genes for markers of several disorders, including Gaucher disease. This ... Read more

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