News

February 14, 2017

Researchers ID Pre-Symptomatic Early Events in Neurological Forms of Gaucher’s

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Structural changes, including the abnormal distribution of lysosomes, were detected in neurons of a mouse model of Gaucher disease (GD) and identified as early signs preceding disease symptoms, a study ... Read more

February 9, 2017

Small Molecule Chaperones Could Be Therapy Path for Gaucher as Well as Parkinson’s

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Small molecule chaperone therapies may play an increasingly important role in the treatment of lysosomal storage disorders such as Gaucher disease, according to a review published in the journal Neural ... Read more

February 7, 2017

NIH-Funded Screening Device for Detection of Gaucher, Other LSDs in Newborns Wins FDA Clearance

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A new screening device called the Seeker System, which detects lysosomal storage disorders (LSDs) like Gaucher disease in newborns, has received de novo clearance from the U.S. Food and Drug ... Read more

February 2, 2017

Adverse Events in Gaucher Patients Taking Cerdelga Occasional and Not Severe, Study Shows

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An analysis of four clinical trials testing Cerdelga (eliglustat) for Gaucher disease patients shows the treatment is safe and without serious adverse events. The study, “A pooled analysis of adverse ... Read more

January 31, 2017January 31, 2017

CHMP Adopts Positive Opinion of Marketing Yargesa to Treat Adults with Mild-to-Moderate Type 1 Gaucher’s

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The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a marketing authorization for Yargesa (miglustat), for the treatment of adults with ... Read more

January 26, 2017

Higher Levels of Autoantibodies in Gaucher Patients Not Linked to Autoimmune Diseases, Study Finds

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A multicenter study shows that people with type 1 Gaucher disease (GD) may have higher levels of serum autoantibodies, but these patients showed no clinical signs of autoimmune disorders. The ... Read more

January 24, 2017

Combination of Zavesca and Enzyme Replacement Therapy Improved Neurological Symptoms in Child with Gaucher Disease

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Combining oral Zavesca (miglustat) with intravenous enzyme replacement therapy (ERT) may prevent neurological problems in patients with neuronopathic Gaucher disease, a case study suggests. The research, “Combination Therapy In A ... Read more

January 19, 2017

Potential Gaucher Treatment Lucerastat Well Tolerated in Early Clinical Trials

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The drug compound lucerastat is well-tolerated overall and does not seem to cause any serious adverse side effects according to a study conducted by scientists at Actelion Pharmaceuticals Ltd.

January 17, 2017

Noninvasive Elastography May Help in Diagnosis of Gaucher Disease Patients

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Noninvasive techniques measuring shear wave velocity in body tissues show potential at distinguishing Gaucher disease patients from other diseases with common symptoms, such as enlarged spleen. The study, “Are transient and shear ... Read more

January 13, 2017

GD Patients Reach Expected Height Regardless of Enzyme Replacement Therapy, Study Shows

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Enzyme replacement therapy (ERT) in Gaucher disease patients has no significant impact on patients’ final height, suggests a small group retrospective study. The study, “Growth and final height of children ... Read more