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Journal of Neuromuscular Diseases|June 13, 2022
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular MyopathyJames J Dowling, Wolfgang Müller-Felber, Barbara K Smith, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|October 15, 2019
Diagnostic utility of transcriptome sequencing for rare Mendelian diseasesHane Lee, Alden Y Huang, Lee-Kai Wang, et al.
Annals of Neurology|November 18, 2017
Natural history of infantile-onset spinal muscular atrophyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.
Annals of Clinical and Translational Neurology|February 23, 2016
Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker studyStephen J Kolb, Christopher S Coffey, Jon W Yankey, et al.
The Lancet. Neurology|April 17, 2024
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trialRabi Tawil, Kathryn R Wagner, Johanna I Hamel, et al.
Ebiomedicine|December 12, 2023
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trialMichael W Lawlor, Benedikt Schoser, Marta Margeta, et al.
Contemporary Clinical Trials|April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophyMichela Guglieri, Kate Bushby, Michael P McDermott, et al.
Neuromuscular Disorders : NMD|September 26, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophyJodi M Wolff, Nora Capocci, Evrim Atas, et al.
Trials|May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trialRebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.
Neurology|February 9, 2024
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled TrialUtkarsh J Dang, Jesse M Damsker, Michela Guglieri, et al.
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