Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
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

Roger L Kaspar  1
Affiliations
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.

PubMed Disclaimer

LinkOut - more resources