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 European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) underscored that the proposal only…
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 does the work of damaged parts of the inner ear (cochlea) to provide sound signals to the brain. Doctors report…
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 behavioral phenotype in Gaucher disease type 3,” was published in the journal Neurology Genetics. Patients with Gaucher disease type 3…
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 Type 1 Gaucher disease (GD), a literature review from 1995 to 2016 found. Elelyso therapy in pediatric patients showed…
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 at New York’s Pierre Hotel, recognize two groups: the employees of UnitedHealth Group who for more than 10…
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 (imiglucerase) in 1991. After analyzing clinical characteristics of…
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. The study, “Parkinson disease in Gaucher disease,” was published in the Journal of Clinical Movement Disorders. Gaucher disease…
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 is enzyme replacement therapy administrated by intravenous infusion. It will begin making Cerdelga available later this year. A medical advisory…
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 of Cerdelga to treat patients who receive standard care with enzyme replacement therapy (ERT) but still present treatment-resistant symptoms.
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 new study from Spain. Researchers say that in Spain, efforts toward early diagnosis have decreased the mean age at diagnosis…
