Natural history of infantile‐onset spinal muscular atrophy
Annals of Neurology2017Vol. 82(6), pp. 883–891
Citations Over TimeTop 1% of 2017 papers
Stephen J. Kolb, Christopher S. Coffey, Jon Yankey, Kristin J. Krosschell, W. David Arnold, Seward B. Rutkove, Kathryn J. Swoboda, Sandra P. Reyna, Ai Sakonju, Basil T. Darras, Richard Shell, Nancy L. Kuntz, Diana Castro, Julie Parsons, Anne M. Connolly, Claudia A. Chiriboga, Craig M. McDonald, W. Bryan Burnette, Klaus Werner, Mathula Thangarajh, Perry B. Shieh, Erika Finanger, Merit Cudkowicz, Michelle McGovern, D. Elizabeth McNeil, Richard S. Finkel, Susan T. Iannaccone, Edward M. Kaye, Allison Kingsley, Samantha Renusch, Vicki L. McGovern, Xueqian Wang, Phillip G. Zaworski, Thomas W. Prior, Arthur H.M. Burghes, Amy Bartlett, John T. Kissel
Abstract
These data of SMA and control outcome measures delineates meaningful change in clinical trials in infantile-onset SMA. The power and utility of NeuroNEXT to provide "real-world," prospective natural history data sets to accelerate public and private drug development programs for rare disease is demonstrated. Ann Neurol 2017;82:883-891.
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