Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery

Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery. Jill Wood, Lori Sames, Allison Moore, Sean Ekins. Drug Discovery Today, Available online 19 August 2013. http://dx.doi.org/10.1016/j.drudis.2013.08.006

Keywords: Advocacy; Collaboration; Crowdfunding; Drug discovery; Foundations; Hereditary diseases; Patient advocates; Patient support; Rare diseases; Translational research; Ultra-rare diseases