Glycogen storage disease type II medical therapy: Difference between revisions
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__NOTOC__ | __NOTOC__ | ||
{{Glycogen storage disease type II}} | {{Glycogen storage disease type II}} | ||
{{CMG}} | {{CMG}}; {{AE}} | ||
==Overview== | ==Overview== | ||
==Medical Therapy== | ==Medical Therapy== | ||
Cardiac and respiratory complications are treated symptomatically. Physical and occupational therapy may be beneficial for some patients. Alterations in diet may provide temporary improvement but will not alter the course of the disease. Genetic counseling can provide families with information regarding risk in future pregnancies. | Cardiac and respiratory complications are treated symptomatically. Physical and occupational therapy may be beneficial for some patients. Alterations in diet may provide temporary improvement but will not alter the course of the disease. Genetic counseling can provide families with information regarding risk in future pregnancies. | ||
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==References== | ==References== | ||
{{reflist|2}} | {{reflist|2}} | ||
[[Category:Endocrinology]] | [[Category:Endocrinology]] | ||
[[Category:Hepatology]] | [[Category:Hepatology]] | ||
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{{WH}} | |||
Revision as of 14:55, 19 July 2016
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Glycogen storage disease type II Microchapters |
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Differentiating Glycogen storage disease type II from other Diseases |
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Glycogen storage disease type II medical therapy On the Web |
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American Roentgen Ray Society Images of Glycogen storage disease type II medical therapy |
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Glycogen storage disease type II medical therapy in the news |
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Directions to Hospitals Treating Glycogen storage disease type II |
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Risk calculators and risk factors for Glycogen storage disease type II medical therapy |
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [3]; Associate Editor(s)-in-Chief:
Overview
Medical Therapy
Cardiac and respiratory complications are treated symptomatically. Physical and occupational therapy may be beneficial for some patients. Alterations in diet may provide temporary improvement but will not alter the course of the disease. Genetic counseling can provide families with information regarding risk in future pregnancies.
On April 28, 2006 the US Food and Drug Administration approved a biologics license application (BLA) for Myozyme (alglucosidase alfa, rhGAA), the first treatment for patients with Pompe disease primarily developed by Dr. Yuan-Tsong Chen (陳垣崇) while he was at Duke University (Dr. Chen is currently the director of the Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan). Myozyme falls under the FDA Orphan Drug designation and was approved under a priority review. Myozyme is manufactured by Genzyme Corp. in Cambridge, MA, USA. FDA Approval News for Myozyme
The FDA approved Myozyme for administration by intravenous infusion of solution into a vein. The safety and efficacy of Myozyme were assessed in two separate clinical trials in 39 infantile-onset patients with Pompe disease ranging in age from 1 month to 3.5 years at the time of the first infusion.
On August 14, 2006, Health Canada approved Myozyme for the treatment of Pompe disease. On June 14, 2007 the Canadian Common Drug Review issued their recommendations regarding public funding for Myozyme therapy. Their recommendation was to provide funding to treat a very small subset of Pompe patients (Infants less one year of age with Cardiomyopathy).[1] The vast majority of developed countries are providing access to therapy for all diagnosed Pompe patients.[2]