TOP NEWS
PEARL Trial-PrEnAtal enzyme Replacement for Lysosomal storage diseases
University of California - San Francisco (UCSF) is excited to announce the rebranding of their phase 1 clinical trail of In Utero Enzyme Replacement Therapy as the PEARL Trial-PrEnAtan enzyme Rreplacement for Llysosomal storage diseases. "We have rebranded our...
OTHER NEWS
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 alfa) for the treatment of Pompe disease. 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 trial of its investigational drug AT2220 (1-deoxynojirimycin HCI) for the treatment of Pompe Disease 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 alfa) 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 alfa) 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
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”
IPA FAQs About the Myozyme Supply Problem
A compilation of the most pertinent information regarding the Myozyme supply issue. The IPA has done its best to gather the most relevant and commonly asked questions regarding this matter. Please download the pdf file. Download complete article
AMDA Statement Regarding the Management of “Myozyme” supply
The AMDA is writing to our Pompe patient community to let everyone know that the AMDA has been working closely with the IPA and Genzyme in order to get communication out to our patient population about the status of the Myozyme supply issue. Download complete...
Acid Maltase Deficiency Association
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