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Neurology|April 5, 2013
Severe congenital RYR1-associated myopathy: the expanding clinicopathologic and genetic spectrumDiana Xerxes Bharucha-Goebel, Mariarita Santi, Livija Medne, et al.
Journal of Multidisciplinary Healthcare|September 3, 2024
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy TherapiesRyan Fischer, Pat Furlong, Annie Kennedy, et al.
Muscle & Nerve|February 8, 2016
Amifampridine phosphate (Firdapse(®)) is effective and safe in a phase 3 clinical trial in LEMSShin J Oh, Natalya Shcherbakova, Anna Kostera-Pruszczyk, et al.
Journal of Neuromuscular Diseases|April 5, 2018
Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn ScreeningJacqueline Glascock, Jacinda Sampson, Amanda Haidet-Phillips, 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.
Therapeutic Advances in Neurological Disorders|July 7, 2025
Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label studyMiriam Freimer, Urvi Desai, Raghav Govindarajan, et al.
Journal of Neuromuscular Diseases|August 23, 2021
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen TreatmentOlga Mitelman, Hoda Z Abdel-Hamid, Barry J Byrne, et al.
Annals of Neurology|February 22, 2026
Electrical Impedance Myography Detects Disease Progression over 12 to 24 Months in Facioscapulohumeral Muscular DystrophyKarlien Mul, Michael P McDermott, Russell J Butterfield, et al.
Muscle & Nerve|April 25, 2020
The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemicAravindhan Veerapandiyan, Kathryn R Wagner, Susan Apkon, et al.
Journal of Neuromuscular Diseases|February 11, 2020
RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study DesignRichard S Finkel, John W Day, Darryl C De Vivo, et al.
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