A generalizable pre-clinical research approach for orphan disease therapy. Review.
Beaulieu CL, Samuels ME, Ekins S, McMaster CR, Edwards AM, Krainer AR, Hicks GG, Frey BJ, Boycott KM, MacKenzie AE. Orphanet Journal of Rare Diseases 2012, 7:39 (15 June 2012).
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