Scalable 9-Step Synthesis of the Splicing Modulator NVS-SM2.

J Org Chem

Laboratory of Therapeutic Innovation (UMR 7200), University of Strasbourg-CNRS, Faculty of Pharmacy , 67401 Illkirch, France.

Published: March 2018

NVS-SM2, the first activator of pre-mRNA splicing, displays remarkable pharmacological in vivo activities in models of spinal muscular atrophy. Herein we describe an improved approach to the synthesis of this compound, which features a convenient introduction of sterically encumbered amine moiety onto a fluoropyridazine intermediate.

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http://dx.doi.org/10.1021/acs.joc.7b03009DOI Listing

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