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Annals of Neurology|August 11, 2017
Natural history of Charcot-Marie-Tooth disease during childhoodKayla M D Cornett, Manoj P Menezes, Rosemary R Shy, et al.Journal of Neuromuscular Diseases|March 13, 2023
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)Richard S Finkel, Basil T Darras, Jerry R Mendell, et al.Neurology|August 8, 2014
Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRSIshu Arpan, Rebecca J Willcocks, Sean C Forbes, et al.Annals of Neurology|February 27, 2013
LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophyKevin M Flanigan, Ermelinda Ceco, Kay-Marie Lamar, et al.Journal of Neuromuscular Diseases|January 22, 2024
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE RegistryLaurent Servais, John W Day, Darryl C De Vivo, et al.Plos One|September 10, 2014
Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with Duchenne muscular dystrophy: a multicenter cross sectional studySean C Forbes, Rebecca J Willcocks, William T Triplett, et al.Annals of Neurology|February 20, 2016
Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohortRebecca J Willcocks, William D Rooney, William T Triplett, et al.Lancet (London, England)|March 13, 2022
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trialCraig M McDonald, Eduardo Marbán, Suzanne Hendrix, et al.BMJ Neurology Open|April 1, 2026
Peripheral frataxin levels govern long-term clinical progression in Friedreich ataxiaChristian Rummey, Ian A Blair, Clementina Mesaros, et al.Muscle & Nerve|February 2, 2019
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophyAnne M Connolly, Craig M Zaidman, Paul T Golumbek, et al.Pageof 38