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Frontiers in Human Neuroscience|June 20, 2022
Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular DystrophyInes Vandekerckhove, Marleen Van den Hauwe, Nathalie De Beukelaer, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|August 18, 2012
Homozygosity for aquaporin 7 G264V in three unrelated children with hyperglyceroluria and a mild platelet secretion defectChristophe Goubau, Jaak Jaeken, Elena N Levtchenko, et al.Annals of Neurology|May 4, 2004
Desmin-related myopathy with Mallory body-like inclusions is caused by mutations of the selenoprotein N geneAna Ferreiro, Chantal Ceuterick-de Groote, Jared J Marks, et al.Lancet (London, England)|April 25, 2015
Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trialGunnar M Buyse, Thomas Voit, Ulrike Schara, et al.Frontiers in Physiology|November 7, 2022
Instrumented strength assessment in typically developing children and children with a neural or neuromuscular disorder: A reliability, validity and responsiveness studyIneke Verreydt, Ines Vandekerckhove, Elze Stoop, et al.Journal of Neuromuscular Diseases|September 23, 2025
Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulationFrancesco Muntoni, James Signorovitch, Michaela Johnson, et al.European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|August 9, 2020
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophyJanbernd Kirschner, Nina Butoianu, Nathalie Goemans, et al.Journal of Neuromuscular Diseases|December 24, 2022
Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1Craig M McDonald, Oscar H Mayer, Kan N Hor, et al.Neurology|October 4, 2017
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophyRonald G Victor, H Lee Sweeney, Richard Finkel, et al.Journal of Neuromuscular Diseases|June 14, 2021
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI TrialCraig M McDonald, Perry B Shieh, Hoda Z Abdel-Hamid, et al.Pageof 11