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Neuromuscular Disorders : NMD|November 28, 2020
Long-term follow-up of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) treated with olesoxime in the OLEOS trialFrancesco Muntoni, Enrico Bertini, Giacomo Comi, et al.BMC Neurology|April 1, 2021
Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part studyTina Duong, Jessica Braid, Hannah Staunton, et al.Neurological Sciences : Official Journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology|September 29, 2022
Use of MFM-20 to monitor SMA types 1 and 2 patients treated with nusinersenLaure Le Goff, Andreea Seferian, Aurelie Phelep, et al.Neurology|August 31, 2018
Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort studyKarolina Aragon-Gawinska, Andreea M Seferian, Aurore Daron, et al.The Lancet. Neurology|May 3, 2017
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trialEnrico Bertini, Eric Dessaud, Eugenio Mercuri, et al.The Journal of Neuroscience Nursing : Journal of the American Association of Neuroscience Nurses|June 9, 2020
Assessing Motor Function in Congenital Muscular Dystrophy Patients Using AccelerometryTokunbor A Lawal, Joshua J Todd, Jeffrey S Elliott, et al.Journal of Neuromuscular Diseases|July 8, 2025
REGISTRE SMA FRANCE: A nationwide observational registry of patients with spinal muscular atrophy in FranceLamiae Grimaldi, Rocio Garcia-Uzquiano, Marta Gomez-Garcia de la Banda, et al.Muscle & Nerve|February 23, 2017
Electrical impedance myography in individuals with collagen 6 and laminin α-2 congenital muscular dystrophy: a cross-sectional and 2-year analysisCarmel Nichols, Minal S Jain, Katherine G Meilleur, et al.European Journal of Neurology|July 15, 2022
Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatmentEugenio Mercuri, Giovanni Baranello, Odile Boespflug-Tanguy, et al.Journal of Neurology|February 3, 2023
Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)Maryam Oskoui, John W Day, Nicolas Deconinck, et al.Pageof 8