Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced that it has initiated a Phase 2 clinical trialA research study that tests new treatments or approaches in people. More of AT2220 (1-deoxynojirimycin HCl),...

We would like to take this opportunity to provide an important update related to MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) manufacturing in the United States.  The FDA has informed Genzyme of its decision on the application for the larger scale manufacturing process to...

In April 2007, Genzyme implemented measures to manage the limited supply of 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 in order to ensure uninterrupted treatment for as many patients as possible.  Since April, both the patient and physician communities have...

CAMBRIDGE, Mass. – Genzyme Corp. (Nasdaq: GENZ) announced today that its Late Onset Treatment Study (LOTS) of Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) met its co-primary efficacy endpoints.  The study was undertaken to evaluate the safety and efficacy of MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More in juvenile and...

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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 would like to take this opportunity to provide a brief update to the Pompe patient community regarding the US supply of Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More).  As planned, Genzyme submitted the necessary documentation to the FDA this month for the licensure of the...

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...

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...

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...