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About Pompe Disease
Pompe disease manifests as a broad spectrum of clinical symptoms. All patients typically experience progressive muscle weakness and breathing difficulty, but the rate of disease progression can vary widely depending on the age of onset and the extent of organ involvement. When symptoms appear within a few months of birth, babies frequently display a markedly enlarged heart and die within the first year of life. When symptoms appear during childhood, adolescence or adulthood, patients may experience steadily progressive debilitation and premature mortality due to respiratory failure. They often require mechanical ventilation to assist with breathing and wheelchairs to assist with mobility.
About Myozyme
Myozyme (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency). The U.S. product label includes a boxed warning with information on the potential risk of hypersensitivity reaction. Life- threatening anaphylactic reactions, including anaphylactic shock, have been observed in patients during Myozyme infusion. Because of the potential for severe infusion reactions, appropriate medical support measures should be readily available when Myozyme is administered. Full prescribing information for the product, including a complete list of the most common adverse reactions, is available on Genzyme's website: http://www.genzyme.com/components/highlights/mz_pi.pdf
About Genzyme
One of the world's leading biotechnology companies, Genzyme is
dedicated to making a major positive impact on the lives of people with
serious dise
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