Challenges in developing therapies for rare diseases including pachyonychia congenita.
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.
Kaspar
Roger L
RL
Transderm and SomaGenics, Inc., Santa Cruz, California, USA. rkaspar@somagenetics.com
D003365:Costs and Cost Analysis; D007644:Darier Disease / Q000628:therapy; D004476:Ectodermal Dysplasia / Q000628:therapy*; D006801:Humans; D007645:Keratoderma, Palmoplantar / Q000628:therapy*; D009264:Nails, Malformed / Q000628:therapy*; D009965:Orphan Drug Production / Q000191:economics; D012106:Research / Q000191:economics; D012109:Research Support as Topic; D012922:Social Change