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Pompe disease News

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 Expands Approval of Lumizyme to treat Pompe Disease Patients of All Ages

Posted 6 Aug 2014 by Drugs.com

August 1, 2014 – The U.S. Food and Drug Administration today announced the approval of Lumizyme (alglucosidase alfa) for treatment of patients with infantile-onset Pompe disease, including patients who are less than 8 years of age. In addition, the Risk Evaluation and Mitigation Strategy (REMS) known as the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program is being eliminated. ...

FDA Approves Lumizyme for Late-Onset Pompe Disease

Posted 28 May 2010 by Drugs.com

SILVER SPRING, Md., May 25 /PRNewswire-USNewswire/ – The U.S. Food and Drug Administration approved Lumizyme (alglucosidase alfa) for patients ages 8 years and older with late-onset (non-infantile) Pompe disease, a rare genetic disorder. Pompe disease occurs in an estimated 1 in every 40,000 to 300,000 births. Its primary symptom is heart and skeletal muscle weakness, progressing to respiratory ...

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Further Information

Related Condition Support Groups

Metabolic Disorder Including Congenital

Related Drug Support Groups

Lumizyme, alglucosidase alfa, Myozyme