TOP NEWS
2024 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More research. This grant opportunity was made possible by the 2024 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
OTHER NEWS
Myozyme Produced at the 2000 L Bioreactor Scale to Receive Accelerated Approval
November 2008 Pompe Program Update
Genzyme Program Update November 10th, 2008 Genzyme would like to take this opportunity to provide an update to the Pompe Community. The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October 21,...
New video added to “Meet the Patients”
New video added to “Meet the Patients”
IPA FAQs About the Myozyme Supply Problem
A compilation of the most pertinent information regarding the MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More 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
AMDA Statement Regarding the Management of “Myozyme” supply
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 MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More supply issue. Download complete...
PRESS RELEASE Priority access for children during Myozyme supply shortage
The European Medicines Agency’s (EMEA) Committee for Medicinal Products (CHMP) has recommended that infants, children and adolescents be given priority access 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), from Genzyme, during the expected supply shortage of the medicine over the...
IPA Statement Regarding the Management of Myozyme Supply
Since the first market approval of MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More, the IPA has liaised directly with Genzyme LSDA group of diseases in which substances build up inside lysosomes because the body cannot break them... More 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...
IPA Statement Regarding the Management of Myozyme Supply – Advice To Pompe Patients
Since the first market approval of MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More, the IPA has liaised directly with Genzyme LSDA group of diseases in which substances build up inside lysosomes because the body cannot break them... More 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...
Guidance to the Pompe Community on the Management of Myozyme Supply
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...
MYOZYME® (2000 L) Supply Update – US Version – January 2009
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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More), Genzyme initiated manufacturing at the 160 liter (L) scale, and has since scaled-up the...
Acid Maltase Deficiency Association
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