News

July 6, 2017

FDA, EMA Propose Exploring Innovative Solutions for Gaucher Disease Drug Development

News

Regulatory agencies in the U.S. and Europe have joined forces to develop a proposal with the aim to spur innovative approaches in the development of new Gaucher disease treatments. The ... Read more

June 29, 2017

Hearing Loss in Gaucher Disease Type 1 Patient Improved with Cochlear Implants

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A young boy with Gaucher disease type 1 and hearing loss improved his hearing after receiving cochlear implants in both ears. A cochlear implant is an electronic medical device that ... Read more

June 27, 2017June 27, 2017

Excessive Anger, Aggressiveness Are Behavioral Symptoms of Gaucher Disease Type 3, Study Finds

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Abnormal behavioral patterns, including increased aggressiveness and anger, can be symptoms of Gaucher disease (GD) type 3 and also markers of disease progression, according to researchers. The study, “Previously unrecognized ... Read more

June 20, 2017June 20, 2017

Taliglucerase Alfa Therapy Seen as Safe and Effective for Children with Gaucher Disease Type 1, Review Finds

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The plant–based enzyme Elelyso (taliglucerase alfa), developed by Protalix and Pfizer as an intravenous enzyme replacement therapy (ERT), is seen as effective and safe for the treatment of children with ... Read more

June 15, 2017June 15, 2017

Gaucher Initiative in Egypt, UnitedHealth Group Recognized at Project HOPE’s Annual NYC Gala

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Project HOPE has named UnitedHealth Group and the Gaucher Initiative in Egypt the recipients of its 2017 Global Health Awards. The awards, handed out during the nonprofit organization’s June 13 annual gala ... Read more

June 13, 2017June 13, 2017

Introduction of Enzyme Replacement Therapy with Cerezyme Has Significantly Decreased Severe GD1 Complications, Study Shows

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The presence of severe complications of Gaucher disease type 1 (GD1) at the time a patient starts treatment has steadily dropped since the introduction of enzyme replacement therapy with Cerezyme ... Read more

June 8, 2017

Different Mutations in Gaucher Disease May Be Linked to Development of Parkinson’s

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New research reports that the difference in severity of Parkinson’s symptoms in patients with Gaucher disease may be due to the difference in the genetic makeup of cells in individuals. ... Read more

June 1, 2017

England’s National Health Service to Make Cerdelga Available as Gaucher Therapy

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Cerdelga (eliglustat) will become the first oral treatment that England’s National Health Service (NHS) makes available to patients with Gaucher disease. At the moment, the only treatment the health service covers ... Read more

May 23, 2017May 23, 2017

Cerdelga Improves Gastrointestinal Symptoms in GD Patient, Korean Case Report Shows

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Cerdelga (eliglustat tartrate) can be effective in treating gastrointestinal lesions in patients with Gaucher disease (GD), according to a clinical case published in BMC Medical Genetics. This finding supports the use ... Read more

May 18, 2017

Earlier Diagnosis of Gaucher in Children Reduces Incidence of Severe Disease, Study Says

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The early diagnosis of children with Gaucher disease eligible for enzyme replacement therapy (ERT) helps decrease the incidence of severe complications and allows patients to have improved development, according to a ... Read more