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Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). [4] [5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase ...
In August 2006, Health Canada approved Myozyme for the treatment of Pompe disease. In June 2007, the Canadian Common Drug Review issued its recommendations regarding public funding for Myozyme therapy. Their recommendation was to provide funding to treat a tiny subset of Pompe patients (Infants less one year of age with cardiomyopathy). [10]
The volume of distribution of avalglucosidase alfa was 3.4 L in patients who had Pompe disease of a late onset. [12] The average half-life of avalglucosidase alfa was 1.6 hours, measured in patients with late stage Pompe disease. [12] There is little information available on the metabolism of the avalglucosidase alfa.
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In this treatment, the accumulated compounds are inhibited from forming in the body of a patient with a lysosomal storage disease. [10] The accumulated compounds are responsible for the symptoms of these disorders, and they form via a multi-step biological pathway. [ 10 ]
William Canfield is a glycobiologist, chief scientific officer and founder of an Oklahoma City-based biotechnology company, Novazyme, which was acquired by Genzyme in August 2001 and developed, among other things, an enzyme that can stabilize (but not cure) Pompe disease, based on Canfield's ongoing research since 1998.
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