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Gaucher's disease |
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Brady's first discovery led directly to Ceredase, a drug for treating the devastating symptoms of Gaucher's Disease, which afflicts fewer than 10,000 people worldwide, generally killing them before they reach adulthood. Last month, the European Union approved a version of NB-DNJ called miglustat for Gaucher's disease, a debilitating genetic disorder. The recent arrival on the market of a modified glucocerebrosidase (Ceredase) for Gaucher's Disease and last year's approval of PEG-adenosine deaminase for severe combined immunodeficiency disease illustrate the growing capabilities of the pharmaceutical industry to provide endogenous compounds that patients are lacking. |
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