TOP NEWS
Essential Update: New Treatment Approaches for Late-Onset Pompe Disease
The AMDA is partnering with Medlive to bring this exciting program to HCPs in the Pompe community. Join leading experts Dr. Tahseen Mozaffar and Dr. Barbara Burton as they discuss late-onset Pompe disease (LOPD). Hear the latest advancements, including newly approved...
OTHER NEWS
Pompe Disease: The Real Story
To read a factual account of the true history of Pompe Disease, 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 Alfa Temporary Access Program (ATAP) and the application for Lumizyme™ (alglucosidase alfa) approval in the United States. In a press release dated December 3,...
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.
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...
Acid Maltase Deficiency Association
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