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Neurology|August 1, 2014
Observational study of spinal muscular atrophy type I and implications for clinical trialsRichard S Finkel, Michael P McDermott, Petra Kaufmann, et al.
Journal of Neurology, Neurosurgery, and Psychiatry|November 29, 2014
CMT subtypes and disease burden in patients enrolled in the Inherited Neuropathies Consortium natural history study: a cross-sectional analysisV Fridman, B Bundy, M M Reilly, et al.
Journal of Neuromuscular Diseases|June 14, 2021
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD TrialRichard S Finkel, Craig M McDonald, H Lee Sweeney, et al.
Microbiology Resource Announcements|November 6, 2020
Genome Sequences of Microbacteriophages Zada and IoannesRazan El Yaman, Jayla S Anderson, Tania M Anderson, et al.
Muscle & Nerve|July 6, 2023
Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE studyThomas O Crawford, Kathryn J Swoboda, Darryl C De Vivo, et al.
Plos One|June 27, 2018
Ambulatory function in spinal muscular atrophy: Age-related patterns of progressionJacqueline Montes, Michael P McDermott, Elizabeth Mirek, et al.
Lancet (London, England)|July 22, 2017
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trialCraig M McDonald, Craig Campbell, Ricardo Erazo Torricelli, et al.
Annals of Neurology|October 7, 2022
Disease Progression in Charcot-Marie-Tooth Disease Related to MPZ Mutations: A Longitudinal StudyVera Fridman, Stefan Sillau, Jacob Bockhorst, et al.
Neuromuscular Disorders : NMD|July 16, 2010
Clinical and genetic characterization of manifesting carriers of DMD mutationsPayam Soltanzadeh, Michael J Friez, Diane Dunn, et al.
Plos Medicine|September 21, 2020
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension studyEdward C Smith, Laurie S Conklin, Eric P Hoffman, et al.
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