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Gaucher Disease News

FDA OKs 1st At-Home Genetic Tests for 10 Disorders

Posted 6 Apr 2017 by Drugs.com

THURSDAY, April 6, 2017 – The U.S. Food and Drug Administration has approved the first home genetic tests for 10 health risks, including Parkinson's disease and late-onset Alzheimer's. The approval – granted to the California-based company 23andMe Inc. – could help test users make lifestyle choices or spark important discussions with health care providers, the FDA said. "Consumers can now have ...

Newborn Screening Tests Approved

Posted 6 Feb 2017 by Drugs.com

MONDAY, Feb. 6, 2017 – A set of screening tests designed to detect four rare metabolic disorders in newborns has been approved by the U.S. Food and Drug Administration. The "Seeker" system of diagnostics is designed to screen for Mucopolysaccharidosis Type 1, Pompe, Gaucher and Fabry. These are the first tests approved to screen for the inherited disorders, which affect proteins that normally ...

FDA Approves Cerdelga (eliglustat) for Type 1 Gaucher Disease

Posted 19 Aug 2014 by Drugs.com

August 19, 2014 – The U.S. Food and Drug Administration today approved Cerdelga (eliglustat) for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials to collect in the spleen, liver and bone marrow. ...

FDA Medwatch Alert: VPRIV (velaglucerase alfa for injection): Recall - Visible Particulate Matter

Posted 20 Mar 2014 by Drugs.com

ISSUE: Shire Pharmaceuticals announced a voluntary recall in the United States of one batch, packaged into three lots, of VPRIV due to the presence of visible particulate matter, identified as stainless steel and barium sulfate. The particulate matter was found in a small number of vials in the three packaged lots of VPRIV. A Shire investigation identified the particulate matter root cause as the ...

FDA Approves New Orphan Drug Elelyso to Treat a Form of Gaucher Disease

Posted 1 May 2012 by Drugs.com

May 1, 2012 – The U.S. Food and Drug Administration today approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials (lipids) to collect in the spleen, liver, kidneys, and ...

FDA Approves VPRIV to Treat Gaucher Disease

Posted 28 Feb 2010 by Drugs.com

The U.S. Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Without this enzyme, harmful amounts of a certain fatty substance (lipid) can build up in the liver, spleen, bones, bone ...

Replacement Therapy Approved for Gaucher Disease

Posted 27 Feb 2010 by Drugs.com

FRIDAY, Feb. 26 – Velaglucerase alfa for injection (VPRIV) has been approved by the U.S. Food and Drug Administration to treat a rare inherited disorder called Gaucher disease, the agency said Friday. The disorder is caused by lack of an enzyme called glucocerebrosidase. This allows a fatty substance called a lipid to build up in the liver, spleen, bones, bone marrow and nervous system. The ...

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Further Information

Related Condition Support Groups

Cerebral Sphingolipidosis, Metabolic Disorder Including Congenital

Related Drug Support Groups

Zavesca, imiglucerase, miglustat, velaglucerase alfa, taliglucerase alfa, eliglustat, Cerezyme, VPRIV, Elelyso, Cerdelga