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A compilation of the most pertinent information regarding the Myozyme supply issue. The IPA has done its best to gather the most relevant and commonly asked questions regarding this matter. Please download the pdf file. Download complete article

The AMDA is writing to our Pompe patient community to let everyone know that the AMDA has been working closely with the IPA and Genzyme in order to get communication out to our patient population about the status of the Myozyme supply issue. Download complete...

The European Medicines Agency’s (EMEA) Committee for Medicinal Products (CHMP) has recommended that infants, children and adolescents be given priority access to Myozyme (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.), from Genzyme, during the expected supply shortage of the medicine over the...

Since the first market approval of Myozyme, the IPA has liaised directly with Genzyme LSDA group of diseases in which substances build up inside lysosomes because the body cannot break them down properly. Therapeutics to insist that an adequate inventory of the product was kept to ensure continuous supply to all patients receiving the treatment; whether through their commercial or...

Since the first market approval of Myozyme, the IPA has liaised directly with Genzyme LSD Therapeutics to insist that an adequate inventory of the product was kept to ensure continuous supply to all patients receiving the treatment; whether through their commercial or...

This guidance is being distributed to all treating physicians worldwide except those in the European Union (EU). Separate guidance will be sent to European healthcare professionals in accordance with the requirements of the European Medicines Agency (EMEA); we expect...

We would like to take this opportunity to provide an important update to the Pompe Community. Background In the early stages of development of Myozyme® (alglucosidase alfa), Genzyme initiated manufacturing at the 160 liter (L) scale, and has since scaled-up the...

We would like to take this opportunity to provide an important update to the global Pompe Community. Background In the early stages of development of Myozyme® (alglucosidase alfa), Genzyme initiated manufacturing at a small scale, and has since scaled-up the process...

The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase alfa produced at the 2000 L bioreactor scale for the treatment of late onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly.. Prior to approval, the company and the agency first...