Natural History of Infantile-Onset Spinal Muscular Atrophy

Kolb SJ, Coffey CS, Yankey JW, Krosschell KP, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Parsons J, Connolly A, Chiriboga CA, McDonald C, Burnette B, Werner W, Thangarajh M, Shieh P, Finanger E, Cudkowicz M, McGovern MM, McNeil DE, Finkel R, Iannacone ST, Kaye E, Kingsley A, Renusch SR, McGovern VL, Wang X, Zaworski PG, Prior TW, Burghes A, Bartlett AHM, Kissel JT and The NeuroNEXT Clinical Trial Network on behalf of the NN101 SMA Biomarker Investigators