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Nature Biotechnology|March 2, 2010
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMNKevin D Foust, Xueyong Wang, Vicki L McGovern, et al.Human Molecular Genetics|April 11, 2015
SMN deficiency disrupts gastrointestinal and enteric nervous system function in miceSara E Gombash, Christopher J Cowley, Julie A Fitzgerald, et al.Annals of Clinical and Translational Neurology|February 11, 2014
Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of ConceptW David Arnold, Paul N Porensky, Vicki L McGovern, et al.Human Molecular Genetics|July 9, 2018
Mild SMN missense alleles are only functional in the presence of SMN2 in mammalsChitra C Iyer, Kaitlyn M Corlett, Aurélie Massoni-Laporte, et al.Plos One|December 2, 2016
Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN RestorationW David Arnold, Sandra Duque, Chitra C Iyer, et al.Human Genetics|February 22, 2019
Complete sequencing of the SMN2 gene in SMA patients detects SMN gene deletion junctions and variants in SMN2 that modify the SMA phenotypeCorey Ruhno, Vicki L McGovern, Matthew R Avenarius, et al.Nature Medicine|October 5, 2021
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissueGretchen Thomsen, Arthur H M Burghes, Caroline Hsieh, et al.Annals of Neurology|November 18, 2017
Natural history of infantile-onset spinal muscular atrophyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.Annals of Clinical and Translational Neurology|February 23, 2016
Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker studyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.Pageof 3