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Medical management of muscle weakness in Duchenne muscular dystrophy
Rivera SR, Jhamb SK, Abdel-Hamid HZ, et al.
PLoS ONE, 2020, 15, 10
Minimal manifestation status and prednisone withdrawal in the MGTX trial
Lee I, Kuo HC, Aban IB, et al.
Neurology, 2020
Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy
Kay DM, Stevens CF, Parker A, et al.
Genetics in medicine : official journal of the American College of Medical Genetics, 2020
Spinal muscular atrophy care in the COVID-19 pandemic era
Veerapandiyan A, Connolly AM, Finkel RS, et al.
Muscle & Nerve, 2020
The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic
Veerapandiyan A, Wagner KR, Apkon S, et al.
Muscle & Nerve, 2020
Spinal muscular atrophy and Farber disease due to ASAH1 variants: A case report
Lee BH, Mongiovi P, Levade T, et al.
American journal of medical genetics. Part A, 2020
Left ventricular dysfunction in Duchenne muscular dystrophy
James KA, Gralla J, Ridall LA, et al.
Cardiology in the young, 2020
Newborn screening for Duchenne muscular dystrophy in China: follow-up diagnosis and subsequent treatment
Ke Q, Zhao ZY, Griggs R, et al.
World journal of pediatrics : WJP, 2020, 13, 3, p 197
Myasthenia Gravis and Congenital Myasthenic Syndromes
Ciafaloni E
Continuum (Minneapolis, Minn.), 2019, 25, 6, p 1767
Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience
Veerapandiyan A, Eichinger K, Guntrum D, et al.
Muscle & Nerve, 2019
Combination therapy with nusinersen and AVXS-101 in SMA type 1
Lee BH, Collins E, Lewis L, et al.
Neurology, 2019
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy
Ke Q, Zhao ZY, Mendell JR, et al.
World journal of pediatrics : WJP, 2019
Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial
Wolfe GI, Kaminski HJ, Aban IB, et al.
The Lancet. Neurology, 2019, 18, 3, p 259
Health profile of a cohort of adults with Duchenne muscular dystrophy
Pandya S, James KA, Westfield C, et al.
Muscle & Nerve, 2018
Application of the International Classification of Functioning, Disability and Health system to symptoms of the Duchenne and Becker muscular dystrophies
Conway KM, Ciafaloni E, Matthews D, et al.
Disability and rehabilitation, 2018, 40, 15, p 1773
Bone Health and Endocrine Care of Boys with Duchenne Muscular Dystrophy: Data from the MD STARnet
Weber DR, Weber DR, Thomas S, et al.
Journal of Neuromuscular Diseases, 2018, 5, 4, p 497
Diagnostic Accuracy of Phenotype Classification in Duchenne and Becker Muscular Dystrophy Using Medical Record Data
Andrews JG, Andrews JG, Lamb MM, et al.
Journal of Neuromuscular Diseases, 2018, 5, 4, p 481
An Update: Myasthenia Gravis and Pregnancy
Hamel J, Ciafaloni E
Neurologic clinics, 2018, 36, 2, p 355
Evidence in focus: Nusinersen use in spinal muscular atrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology
Michelson D, Ciafaloni E, Ashwal S, et al.
Neurology, 2018, 91, 20, p 923
Cervical puncture to deliver nusinersen in patients with spinal muscular atrophy
Veerapandiyan A, Veerapandiyan A, Pal R, et al.
Neurology, 2018, 91, 7
Implementation of Duchenne Muscular Dystrophy Care Considerations
Andrews JG, Conway K, Westfield C, et al.
Pediatrics, 2018, 142, 1
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Crow RA, Hart KA, McDermott MP, et al.
Trials, 2018, 19, 1
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Guglieri M, Bushby K, McDermott MP, et al.
Contemporary clinical trials, 2017, 58, p 34
Perceived quality of life among caregivers of children with a childhood-onset dystrophinopathy: a double ABCX model of caregiver stressors and perceived resources
Frishman N, Conway KC, Andrews J, et al.
Health and quality of life outcomes, 2017, 15, 1
Secondary Conditions Among Males With Duchenne or Becker Muscular Dystrophy
Latimer R, Street N, Conway KC, et al.
Journal of child neurology, 2017, 32, 7, p 663
