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- W2791602357 abstract "Anyone puzzling over the business case for developing drugs for tiny patient populations need look no further than Genzyme. Founded in Boston in 1981, the biotechnology firm pioneered the model for rare disease drug development that is followed today: make an impact on a previously untreated rare disease, charge high prices, and be rewarded with significant revenues and a long reign in the marketplace. Genzyme made its mark by introducing the first treatment for Gaucher’s disease, a lysosomal storage disease caused by a deficiency in the lipid-busting enzyme glucocerebrosidase. Similar to Hunter syndrome, Gaucher’s occurs when the absence of that key enzyme causes a buildup of molecules in the lysosome and results in a variety of problems. Complications from Gaucher’s include enlarged organs, bone pain, and anemia. When Genzyme embarked on developing an enzyme replacement therapy for type 1 Gaucher’s, which does not affect the central nervous system, the disease ..." @default.
- W2791602357 created "2018-03-29" @default.
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- W2791602357 date "2013-05-13" @default.
- W2791602357 modified "2023-09-25" @default.
- W2791602357 title "The Business Of Rarity" @default.
- W2791602357 doi "https://doi.org/10.1021/cen-09119-cover2" @default.
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