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European Journal of Neurology|June 27, 2025
Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With NusinersenGiorgia Coratti, Francesca Bovis, Marika Pane, et al.Neurology|August 28, 2019
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle functionEric P Hoffman, Benjamin D Schwartz, Laurel J Mengle-Gaw, et al.Journal of Neuromuscular Diseases|March 1, 2024
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3Amy Wolfe, Georgia Stimpson, Danielle Ramsey, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|May 7, 2025
Current clinical applications of AAV-mediated gene therapyBarry J Byrne, Kevin M Flanigan, Susan E Matesanz, et al.Brain : a Journal of Neurology|November 2, 2013
SIL1 mutations and clinical spectrum in patients with Marinesco-Sjogren syndromeMichael Krieger, Andreas Roos, Claudia Stendel, et al.Neuromuscular Disorders : NMD|January 4, 2022
Revised upper limb module in type II and III spinal muscular atrophy: 24-month changesGiorgia Coratti, Maria Carmela Pera, Jacqueline Montes, et al.Brain : a Journal of Neurology|January 8, 2021
Natural history of Charcot-Marie-Tooth disease type 2A: a large international multicentre studyMenelaos Pipis, Shawna M E Feely, James M Polke, 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.Pharmacological Research|September 17, 2018
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drugLaurie S Conklin, Jesse M Damsker, Eric P Hoffman, et al.Neurology|February 13, 2020
A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scoresVera Fridman, Stefan Sillau, Gyula Acsadi, et al.Pageof 38