More on SMN: https://ghr.nlm.nih.gov/gene/SMN2 http://www.curesma.org/sma/causes-diagnoses/genetics/ https://www.bcbsri.com/sites/default/files/polices/Nusinersen%20_for%20_Spinal_Muscular_%20Atrophy%206_2017.pdf https://www.webmd.com/children/news/20171101/gene-therapy-new-drug-fight-rare-disease-in-kids#1 http://techtransfercentral.com/2017/08/29/indiana-u-lands-research-agreement-with-spotlight-innovations-to-develop-new-therapies-for-spinal-muscular-atrophy/