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W. David Arnold, MD
W. David Arnold, MD
NextGen Precision Health, University of Missouri
Verifisert e-postadresse på health.Missouri.edu
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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
23912017
Spinal muscular atrophy: diagnosis and management in a new therapeutic era
WD Arnold, D Kassar, JT Kissel
Muscle & nerve 51 (2), 157-167, 2015
4882015
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
4052017
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
2262019
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
2032019
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
1902019
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
1572015
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
1422016
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
1312010
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
1102018
Multifocal motor neuropathy: a review of pathogenesis, diagnosis, and treatment
VH Lawson, WD Arnold
Neuropsychiatric disease and treatment, 567-576, 2014
1082014
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
1072016
Spinal muscular atrophy: development and implementation of potential treatments
WD Arnold, AHM Burghes
Annals of neurology 74 (3), 348-362, 2013
1052013
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
952021
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
942015
Spinal muscular atrophy
ES Arnold, KH Fischbeck
Handbook of clinical neurology 148, 591-601, 2018
902018
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
882020
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
Journal of visualized experiments: JoVE, 52899, 2015
862015
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, 2013
852013
Base editing rescue of spinal muscular atrophy in cells and in mice
M Arbab, Z Matuszek, KM Kray, A Du, GA Newby, AJ Blatnik, A Raguram, ...
Science 380 (6642), eadg6518, 2023
802023
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