%0 Journal Article %T A generalizable pre-clinical research approach for orphan disease therapy %A Chandree L Beaulieu %A Mark E Samuels %A Sean Ekins %A Christopher R McMaster %A Aled M Edwards %A Adrian R Krainer %A Geoffrey G Hicks %A Brendan J Frey %A Kym M Boycott %A Alex E MacKenzie %J Orphanet Journal of Rare Diseases %D 2012 %I BioMed Central %R 10.1186/1750-1172-7-39 %K Orphan disease therapy %K Preclinical drug development %K Generalizable screening methods %K Translational toolbox %U http://www.ojrd.com/content/7/1/39/abstract