Small Molecule Therapy for Genetic Disease - Thoene, Jess G. (Cambridge : Cambridge University Press, 2010)
Title from publisher's bibliographic system (viewed on 28 Apr 2015).
Keywords: Metabolism, Inborn errors of -- Chemotherapy -- Handbooks, manuals, etc; Metabolism, Inborn errors of -- Gene therapy -- Handbooks, manuals, etc; Genetic disorders -- Chemotherapy -- Handbooks, manuals, etc; Genetic Diseases, Inborn -- drug therapy; Orphan Drug Production; Rare Diseases -- drug therapy; Small Molecule Libraries -- therapeutic use