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imiglucerase : ウィキペディア英語版
imiglucerase

Imiglucerase is a medication used in the treatment of Gaucher's disease.
It is a recombinant DNA-produced analogue of human β-glucocerebrosidase.
Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme Corporation. It is given
intravenously after reconstitution as a treatment for Type 1 Gaucher's disease. It is available in formulations containing 200 or 400 units per vial. The specific activity of highly purified human enzyme is 890,000 units/mg. A typical dose is 2.5U/kg every two weeks, up to a maximum of 60 U/kg once every two weeks, and safety has been established from ages 2 and up. It is one of the most expensive drugs sold, with an annual cost to U.S. patients of $200,000.〔Balancing innovation, access, and profits -- marketing exclusivity for biologics, Alfred B. Engelberg et al., N Engl J Med 361:1917〕 Due to the low profitability (and high expense) of developing medications for rare conditions, imiglucerase has been granted orphan drug status in the USA, Australia, and Japan.
Cerezyme was one of the drugs manufactured at Genzyme's Allston, Massachusetts plant, for which production was disrupted in 2009 after contamination with Vesivirus 2117.
==See also==

*Other drugs for the treatment of Gaucher's disease
*
*Afegostat (development terminated)
*
*Eliglustat
*
*Miglustat

抄文引用元・出典: フリー百科事典『 ウィキペディア(Wikipedia)
ウィキペディアで「imiglucerase」の詳細全文を読む



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