Challenges in developing therapies for rare diseases including pachyonychia congenita

被引：28

作者：

Kaspar, RL

机构：

[1] Transderm, Santa Cruz, CA 95060 USA

[2] SomaGenet Inc, Santa Cruz, CA 95060 USA

来源：

JOURNAL OF INVESTIGATIVE DERMATOLOGY SYMPOSIUM PROCEEDINGS | 2005年 / 10卷 / 01期

关键词：

orphan disease; pachyonychia congenita; rare disease;

D O I：

10.1111/j.1087-0024.2005.10208.x

中图分类号：

R75 [皮肤病学与性病学];

学科分类号：

100206 ;

摘要：

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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收藏

页码：62 / 66

页数：5

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