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Neuromuscular Disorders : NMD|January 7, 2018
Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethicsRichard S Finkel, Eugenio Mercuri, Oscar H Meyer, et al.Lancet (London, England)|July 22, 2017
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trialCraig M McDonald, Craig Campbell, Ricardo Erazo Torricelli, et al.Journal of Child Neurology|March 21, 2012
Consensus statement on standard of care for congenital myopathiesChing H Wang, James J Dowling, Kathryn North, et al.Journal of Comparative Effectiveness Research|August 28, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophyCraig Campbell, Richard J Barohn, Enrico Bertini, et al.Journal of Child Neurology|November 17, 2010
Consensus statement on standard of care for congenital muscular dystrophiesChing H Wang, Carsten G Bonnemann, Anne Rutkowski, et al.Nature Communications|March 29, 2019
Myoglobinopathy is an adult-onset autosomal dominant myopathy with characteristic sarcoplasmic inclusionsMontse Olivé, Martin Engvall, Gianina Ravenscroft, et al.Journal of Neurology|October 29, 2013
Mapping the differences in care for 5,000 spinal muscular atrophy patients, a survey of 24 national registries in North America, Australasia and EuropeCatherine L Bladen, Rachel Thompson, Jacqueline M Jackson, et al.Pageof 8