Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy. - PubMed - NCBI

J Med Chem. 2017 May 25;60(10):4444-4457. doi: 10.1021/acs.jmedchem.7b00406. Epub 2017 May 4.