Challenges in Developing Therapies for Rare Diseases Including Pachyonychia Congenita

The 4th Intercontinental Meeting of Hair Research

Roger L. Kaspar

Others  :  http://www.nature.com/jidsp/journal/v10/n1/pdf/5640178a.pdf
PID  :  25016

来源: CEUR

【 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.