Single-dose gene-replacement therapy for spinal muscular atrophy JR Mendell, S Al-Zaidy, R Shell, WD Arnold, LR Rodino-Klapac, TW Prior, ... New England Journal of Medicine 377 (18), 1713-1722, 2017 | 2142 | 2017 |
Spinal muscular atrophy: diagnosis and management in a new therapeutic era WD Arnold, D Kassar, JT Kissel Muscle & nerve 51 (2), 157-167, 2015 | 426 | 2015 |
Natural history of infantile‐onset spinal muscular atrophy SJ Kolb, CS Coffey, JW Yankey, K Krosschell, WD Arnold, SB Rutkove, ... Annals of neurology 82 (6), 883-891, 2017 | 357 | 2017 |
Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy S Al‐Zaidy, AS Pickard, K Kotha, LN Alfano, L Lowes, G Paul, K Church, ... Pediatric pulmonology 54 (2), 179-185, 2019 | 199 | 2019 |
Impact of age and motor function in a phase 1/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy LP Lowes, LN Alfano, WD Arnold, R Shell, TW Prior, M McColly, ... Pediatric neurology 98, 39-45, 2019 | 181 | 2019 |
AVXS-101 (onasemnogene abeparvovec) for SMA1: comparative study with a prospective natural history cohort SA Al-Zaidy, SJ Kolb, L Lowes, LN Alfano, R Shell, KR Church, ... Journal of neuromuscular diseases 6 (3), 307-317, 2019 | 168 | 2019 |
A large animal model of spinal muscular atrophy and correction of phenotype SI Duque, WD Arnold, P Odermatt, X Li, PN Porensky, L Schmelzer, ... Annals of neurology 77 (3), 399-414, 2015 | 152 | 2015 |
Baseline results of the Neuro NEXT spinal muscular atrophy infant biomarker study SJ Kolb, CS Coffey, JW Yankey, K Krosschell, WD Arnold, SB Rutkove, ... Annals of clinical and translational neurology 3 (2), 132-145, 2016 | 130 | 2016 |
Mutant small heat shock protein B3 causes motor neuropathy: utility of a candidate gene approach SJ Kolb, PJ Snyder, EJ Poi, EA Renard, A Bartlett, S Gu, S Sutton, ... Neurology 74 (6), 502-506, 2010 | 123 | 2010 |
Spinal muscular atrophy: development and implementation of potential treatments WD Arnold, AHM Burghes Annals of neurology 74 (3), 348-362, 2013 | 107 | 2013 |
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy DA Griffin, RW Johnson, JM Whitlock, ER Pozsgai, KN Heller, WE Grose, ... Human molecular genetics 25 (10), 1900-1911, 2016 | 106 | 2016 |
Muscle strength and size are associated with motor unit connectivity in aged mice KA Sheth, CC Iyer, CG Wier, AE Crum, A Bratasz, SJ Kolb, BC Clark, ... Neurobiology of aging 67, 128-136, 2018 | 95 | 2018 |
Multifocal motor neuropathy: a review of pathogenesis, diagnosis, and treatment VH Lawson, WD Arnold Neuropsychiatric disease and treatment, 567-576, 2014 | 92 | 2014 |
The genetics of Charcot–Marie–Tooth disease: current trends and future implications for diagnosis and management JC Hoyle, MC Isfort, J Roggenbuck, WD Arnold The Application of Clinical Genetics, 235-243, 2015 | 89 | 2015 |
Efficient precise in vivo base editing in adult dystrophic mice L Xu, C Zhang, H Li, P Wang, Y Gao, NA Mokadam, J Ma, WD Arnold, ... Nature communications 12 (1), 3719, 2021 | 86 | 2021 |
Electrophysiological biomarkers in spinal muscular atrophy: proof of concept WD Arnold, PN Porensky, VL McGovern, CC Iyer, S Duque, X Li, K Meyer, ... Annals of clinical and translational neurology 1 (1), 34, 2014 | 79 | 2014 |
Electrophysiological motor unit number estimation (MUNE) measuring compound muscle action potential (CMAP) in mouse hindlimb muscles WD Arnold, KA Sheth, CG Wier, JT Kissel, AH Burghes, SJ Kolb JoVE (Journal of Visualized Experiments), e52899, 2015 | 74 | 2015 |
Guidelines on clinical presentation and management of nondystrophic myotonias BC Stunnenberg, S LoRusso, WD Arnold, RJ Barohn, SC Cannon, ... Muscle & nerve 62 (4), 430-444, 2020 | 71 | 2020 |
OATP1B2 deficiency protects against paclitaxel-induced neurotoxicity AF Leblanc, JA Sprowl, P Alberti, A Chiorazzi, WD Arnold, AA Gibson, ... The Journal of clinical investigation 128 (2), 816-825, 2018 | 69 | 2018 |
Defective fast inactivation recovery of Nav1.4 in congenital myasthenic syndrome WD Arnold, DH Feldman, S Ramirez, L He, D Kassar, A Quick, TL Klassen, ... Annals of neurology 77 (5), 840-850, 2015 | 69 | 2015 |