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Scientific Reports|November 29, 2024
Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpointLaurent Servais, Paul Strijbos, Margaux Poleur, et al.Muscle & Nerve|June 27, 2015
Old measures and new scores in spinal muscular atrophy patientsElena Mazzone, Jacqueline Montes, Marion Main, et al.Neurogenetics|September 19, 2014
Unraveling the genetic landscape of autosomal recessive Charcot-Marie-Tooth neuropathies using a homozygosity mapping approachMagdalena Zimoń, Esra Battaloğlu, Yesim Parman, et al.Neuromuscular Disorders : NMD|March 28, 2021
The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophyClaudia Brogna, Giorgia Coratti, Rachele Rossi, et al.Neuromuscular Disorders : NMD|January 19, 2016
Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trialsEugenio Mercuri, Richard Finkel, Jacqueline Montes, et al.The Lancet. Neurology|March 20, 2024
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trialEugenio Mercuri, Juan J Vilchez, Odile Boespflug-Tanguy, 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.Plos One|June 25, 2021
North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow upGiorgia Coratti, Marika Pane, Claudia Brogna, et al.Muscle & Nerve|July 22, 2014
Ataluren treatment of patients with nonsense mutation dystrophinopathyKatharine Bushby, Richard Finkel, Brenda Wong, 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.Pageof 11