TOP NEWS
2023 Helen Walker Grant Recipient
Project title: AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. More control of glycogenA stored form of sugar used for energy. More in skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. More 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
IPA Statement: The End of Myozyme Supply Restrictions
Dear IPA Affiliate, You will no doubt have heard the wonderful news that MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More from the Belgian production facility has been approved for patients within the European Community (see the IPA website for the press release). Another great success has been the adoption...
Pompe Program Update from Genzyme
Supply Update In January 2009, Genzyme notified the Pompe community that we were experiencing a temporary constraint in the global supply of Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) produced at the 2000 L bioreactor scale and that inventories were extremely tight. We are pleased...
Genzyme Receives Complete Response Letter from FDA on Lumizyme Application
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that it has received a complete response letter from the FDA regarding its application to market Lumizyme™ (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) for the treatment of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. In its letter, the agency outlines...
Amicus Therapeutics Suspends Enrollment for Phase 2 Clinical Trial of AT2220 for Pompe
Amicus Therapeutics announced today that the Company has suspended enrollment for the Phase 2 clinical trialA research study that tests new treatments or approaches in people. More of its investigational drug AT2220 (1-deoxynojirimycin HCI) for the treatment of Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More and that it has received verbal notice from the U.S. Food and...
European Authorities Approve Larger-Scale Production of Genzyme’s Myozyme
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that the European Commission has approved the production of Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. The product will be...
Genzyme Receives Positive Opinion on Myozyme from CHMP
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that the Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion on the company’s variation to produce Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) at the 4000 liter (L) bioreactor...
November 2008 Pompe Program Update 2
Genzyme Pompe Program Update- November 17, 2008 We would like to take this opportunity to provide an important update to the Pompe Community. The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase...
Myozyme Produced at the 2000 L Bioreactor Scale to Receive Accelerated Approval
November 2008 Pompe Program Update
Genzyme Program Update November 10th, 2008 Genzyme would like to take this opportunity to provide an update to the Pompe Community. The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October 21,...
New video added to “Meet the Patients”
New video added to “Meet the Patients”
Acid Maltase Deficiency Association
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