TOP NEWS
2023 Helen Walker Grant Recipient
Project title: Autophagy control of glycogen in skeletal muscle This year, the AMDA was fortunate to receive 13 grant applications focusing on both clinical research and basic science. It has been a challenge to select one. Dr. Wyant was chosen because he has embarked...
OTHER NEWS
Genzyme Pompe Program Update—November 2008
The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase alfa produced at the 2000 L bioreactor scale for the treatment of late onset Pompe disease. Prior to approval, the company and the agency first...
Genzyme Pompe Program Update—November 10, 2008
The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October 21, 2008 to discuss Genzyme’s Biologics License Application (BLA) for alglucosidase alfa produced at the 2000 L bioreactor scale to treat...
Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease
Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced that it has initiated a Phase 2 clinical trial of AT2220 (1-deoxynojirimycin HCl),...
Genzyme Pompe Program Update—April 2008
We would like to take this opportunity to provide an important update related to Myozyme (alglucosidase alfa) manufacturing in the United States. The FDA has informed Genzyme of its decision on the application for the larger scale manufacturing process to...
January 2008 US Myozyme Supply Update
In April 2007, Genzyme implemented measures to manage the limited supply of Myozyme® (alglucosidase alfa) in the United States in order to ensure uninterrupted treatment for as many patients as possible. Since April, both the patient and physician communities have...
Genzyme Study of Myozyme® for Late-Onset Pompe Patients
CAMBRIDGE, Mass. – Genzyme Corp. (Nasdaq: GENZ) announced today that its Late Onset Treatment Study (LOTS) of Myozyme® (alglucosidase alfa) met its co-primary efficacy endpoints. The study was undertaken to evaluate the safety and efficacy of Myozyme in juvenile and...
Genzyme Pompe Program Update—October 2007
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MTAP Fact Sheet June 2007
MTAP, the Myozyme® (alglucosidase alfa) Temporary Access Program, is a clinical program to provide access to Myozyme produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Genzyme Pompe Program Update—June 2007
Genzyme would like to take this opportunity to provide a brief update to the Pompe patient community regarding the US supply of Myozyme® (alglucosidase alfa). As planned, Genzyme submitted the necessary documentation to the FDA this month for the licensure of the...
MTAP Fact Sheet May 2007
MTAP, the Myozyme® (alglucosidase alfa) Temporary Access Program, is a clinical program to provide access to Myozyme produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Acid Maltase Deficiency Association
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