Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Autor: De Vivo, Darryl C. a, ⁎, Bertini, Enrico b, Swoboda, Kathryn J. c, Hwu, Wuh-Liang d, Crawford, Thomas O. e, Finkel, Richard S. f, Kirschner, Janbernd g, h, Kuntz, Nancy L. i, Parsons, Julie A. j, Ryan, Monique M. k, Butterfield, Russell J. l, Topaloglu, Haluk m, Ben-Omran, Tawfeg n, o, Sansone, Valeria A. p, q, Jong, Yuh-Jyh r, Shu, Francy s, Staropoli, John F. t, 1, Kerr, Douglas t, 1, Sandrock, Alfred W. t, Stebbins, Christopher t, Petrillo, Marco t, Braley, Gabriel t, Johnson, Kristina t, Foster, Richard u, Gheuens, Sarah t, Bhan, Ishir t, Reyna, Sandra P. t, 1, Fradette, Stephanie t, Farwell, Wildon t
Zdroj: In Neuromuscular Disorders November 2019 29(11):842-856
Databáze: ScienceDirect