TOP NEWS
2024 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 13th Annual PCMA’s Pull for Pompe will take place on Saturday, April 27, 2024 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
OTHER NEWS
FDA Approves Genzyme’s Myozyme® for All Patients with Pompe Disease
Genzyme Corp. (Nasdaq: GENZ) announced today that the Food and Drug Administration has granted marketing approval for Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) in the United States. MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More has been approved for the treatment of patients with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More, a debilitating,...
European Commission Approves Genzyme’s Myozyme®
Product is First Treatment for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Genzyme Corp. (Nasdaq: GENZ) announced today that it has received marketing authorization for Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) in the European Union. MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More has been approved for long-term enzymeA protein that helps the body carry out chemical reactions. More replacement therapy in...
Genzyme Provides Update on U.S. Marketing Application for Myozyme
Genzyme Corp. (Nasdaq: GENZ) announced today that the Food and Drug Administration has extended by 90 days the review period for the biologics license application for Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More). The new action date for the application is April 28, 2006. The purpose...
The IPA Pompe Connections Brochure
The IPA has developed the Pompe connections brochure to help get patients and their families timely and relevant information about Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More and the developments that are happening. Please visit the Pompe Connections Brochure page.
Genzyme Submits BLA for Myozyme® for Treatment of Pompe Disease
Genzyme Corp. (Nasdaq: GENZ) announced today that it has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration for Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More). If approved, MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More would be the first treatment developed for patients with Pompe...
Summary of the 2005 AFM-IPA Conference
May 11th 2005 IPA Conference Pompe’s disease Nantes, France. “Emerging therapies for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More” Dr. Ans van der Ploeg, Erasmus MC Rotterdam the Netherlands, talked about the natural course in late onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More. She informed the audience about the outcome...
Genzyme Reports Interim Results from Pivotal Study of Myozyme
CAMBRIDGE, Mass.—Genzyme Corp. (Nasdaq: GENZ) announced today that it has completed a planned analysis of interim data from its pivotal clinical trialA research study that tests new treatments or approaches in people. More of Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More), which is being studied for the treatment of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. The interim analysis was...
Genzyme Files for European Approval of Myozyme® For Treatment of Pompe Disease
CAMBRIDGE, MA—Genzyme Corp. (Nasdaq: GENZ) announced today that the European Medicines Agency (EMEA) has accepted its marketing authorization application for Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More), an investigational enzymeA protein that helps the body carry out chemical reactions. More replacement therapy for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. If approved,...
Summary of 2004 International Pompe Association Conference
On 9th and 10th October, the AGSD (UK) held its annual patient conference. This year we were more ambitious than usual in that the Pompe Workshop extended into a second day. Families and speakers arrived on the Friday night and we held an informal conference dinner on...
IPA Conference – October 30 – November 1, 2003
Medical experts and patients are invited to attend the International Pompe Conference 2003. The goal of the conference is to bring together scientific expertise and patients’ experience. On the way towards a therapy for Pompe’s Disease there were and are many examples...


