Editorial: Extraordinary Measures or an Extraordinary Tale? (Update)
By: Tiffany House--AMDA President posted on: February 15, 2010 What happened to the TRUTH? In the wake of the recent article by Quest Magazine (January 2010) and the upcoming film “Extraordinary Measures” I feel that I must say something as to the accuracy of the...
Pompe Disease: The Real Story
To read a factual account of the true history of Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart., please visit Pompe Disease—The Real Story.
AMDA Press Release Regarding Extraordinary Measures
The AMDA issued a press release regarding the film, Extraordinary Measures. The press release can be found here. In addition, the AMDA created a one-page fact sheet regarding Pompe disease to help raise awareness of the condition. That fact sheet can be found...
ATAP Inclusion/Exclusion and List of Study Sites
This information was complied from the information provided on clinicaltrials.gov. To read, please download file by clicking here.
December 2009 Pompe Program Update
Genzyme would like to provide an update to the US Pompe community about the status of the Alglucosidase AlfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme. Temporary Access Program (ATAP) and the application for Lumizyme™ (alglucosidase alfa) approval in the United States. In a press release dated December 3,...
Pompe Disease—By a Pompe Patient
By Dawn Harris Kendall posted on: December 01, 2009 Living with Pompe Elizabeth Nunnery takes her infant daughter, Zoe, to the hospital at 8:30 in the morning. A nurse verifies Zoe’s medical information and hooks Zoe up to machines monitoring her blood oxygen level,...
Influenza Vaccinations and Pompe Disease
Guidelines from Erasmus Medical Centre, Rotterdam, the Netherlands As a guideline we recommend that all patients with Pompe disease are vaccinated against influenza. This applies both to the seasonal influenza as well as for the 2009 flu pandemic (influenza virus...
Genzyme invites the Gaucher, Fabry, Pompe and MPS I communities to a Town Hall Meeting
Genzyme Manufacturing Update Genzyme invites all patients to participate in a forum to discuss recent FDA reports and media attention related to manufacturing operations in Allston, Massachusetts. Date: Monday, November 23, 2009 Time: 6:00 PM – 7:00 PM (EST) US Toll...
November 2009 Pompe Program Update from Genzyme
Genzyme would like to provide an update to the US Pompe community about the status of the application for Lumizyme™ (alglucosidase alfa) produced at the 2000 L scale in the United States. To read full Program Update, please click here.
Amicus Therapeutics Announces Plan to Initiate Phase 1 Study of AT2220 for Pompe Disease
CRANBURY, N.J., Sept 30, 2009 - - Amicus Therapeutics today announced it plans to initiate a Phase 1 study of AT2220 (1-deoxynojirimycin HCl), its investigational drug in development for the treatment of Pompe Disease. The primary objective of this study is to...
Genzyme’s Second Quarter Earnings Statement
In their second quarter earning statement, Genzyme provided an update on the situation at the Allston manufacturing plant, and the US Lumizyme application. To read full statement, please click here.
Changing Lives—UTSA Sombrilla
By: Jason B. Johnson posted on: July 20, 2009 Published in University of Texas San Antonio’s Sombrilla Magazine: Summer 2009 Tiffany House was just 11 when she was diagnosed with a rare and often fatal disease called Acid Maltase Deficiency. Doctors told her she...
Genzyme Submits All Information Requested by FDA for Lumizyme
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that it has submitted the final documentation to address all items in the FDA’s complete response letter for LumizymeTM (alglucosidase alfa), produced at the 2,000 L bioreactor scale. The submission...
IPA Statement: The End Of Myozyme Supply Restrictions
By: IPA posted on: May 08, 2009 Dear IPA Affiliate, You will no doubt have heard the wonderful news that Myozyme from the Belgian production facility has been approved for patients within the European Community (see the IPA website for the press release). Another...
IPA Statement: The End of Myozyme Supply Restrictions
Dear IPA Affiliate, You will no doubt have heard the wonderful news that Myozyme 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 alfa) 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 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 trialA research study that tests new treatments or approaches in people. 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...