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
MTAP Fact Sheet May 2007
MTAP, the Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) Temporary Access Program, is a clinical program to provide access to MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Genzyme Pompe Program Update—April 2007
As many of you are aware, a standard part of the biologics drug development process is to incrementally scale-up manufacturing capacity as demand for the product increases. During the development of Myozyme®, Genzyme began the manufacturing process using a smaller...
Letter From IPA and Genzyme Concerning LOTS
Dear IPA members, Pompe patients, family members and GSD-net participants, The IPA board received several questions concerning participation in the LOTS trial. As there are certain restrictions to the market approval of MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More we asked Genzyme to make a written...
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...
Acid Maltase Deficiency Association
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