Elisabetta Soragni & Joel M. Gottesfeld. Expert Opinion on Orphan Drugs, Published online: 31 Jul 2016 DOI:10.1080/21678707.2016.1215910
Expert opinion: 2-aminobenzamide class I HDAC inhibitors are attractive therapeutic small molecules for FRDA. These molecules increase FXN gene expression in human neuronal cells derived from patient induced pluripotent stem cells, and in two mouse models for the disease, as well as in circulating lymphocytes in patients treated in a phase Ib clinical trial. Medicinal chemistry efforts have identified compounds with improved brain penetration, metabolic stability and efficacy in the human neuronal cell model. A clinical candidate will soon be identified for further human testing
Sunday, August 7, 2016
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