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Review
. 2005 Oct;10(1):62-6.
doi: 10.1111/j.1087-0024.2005.10208.x.

Challenges in developing therapies for rare diseases including pachyonychia congenita

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Free article
Review

Challenges in developing therapies for rare diseases including pachyonychia congenita

Roger L Kaspar. J Investig Dermatol Symp Proc. 2005 Oct.
Free article

Abstract

The ability to attract sufficient resources to effectively develop therapeutics for rare diseases is a daunting task. This review summarizes existing resources for rare diseases and discusses some of the challenges and strategies associated with developing therapies for small patient populations with an emphasis on pachyonychia congenita.

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