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Policy alternatives for treatments for rare diseases.

作者信息

Panju Abbas H, Bell Chaim M

机构信息

DeGroote School of Business, McMaster University, Hamilton, Ont.

出版信息

CMAJ. 2010 Nov 23;182(17):E787-92. doi: 10.1503/cmaj.081429. Epub 2010 Jul 12.

Abstract
摘要

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The neurological manifestations of Gaucher disease type 1: the French Observatoire on Gaucher disease (FROG).
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Enzyme therapy for Fabry patients in jeopardy.
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Outpatient gatifloxacin therapy and dysglycemia in older adults.
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Orphan drug development is progressing too slowly.
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Adopting orphan drugs--two dozen years of treating rare diseases.
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