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Neurology. Genetics|September 3, 2025
Study of Testosterone and Recombinant Human Growth Hormone in Facioscapulohumeral Muscular DystrophyChad Rydel Heatwole, Elizabeth Luebbe, Johanna Hamel, et al.
Annals of Clinical and Translational Neurology|June 23, 2023
Newborn screening for Duchenne muscular dystrophy: A two-year pilot studyNorma P Tavakoli, Dorota Gruber, Niki Armstrong, et al.
Neurology and Therapy|January 10, 2026
Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK TrialJerry R Mendell, Francesco Muntoni, Craig M McDonald, et al.
The Lancet. Neurology|September 22, 2023
Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trialPedro M Machado, Michael P McDermott, Thomas Blaettler, et al.
Nature Medicine|October 9, 2024
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trialJerry R Mendell, Francesco Muntoni, Craig M McDonald, et al.
Neurology|December 3, 2021
Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG StudyRichard J Nowak, Christopher S Coffey, Jonathan M Goldstein, et al.
Pediatric Neurology|March 24, 2009
Newborn screening for Krabbe disease: the New York State modelPatricia K Duffner, Michele Caggana, Joseph J Orsini, et al.
Contemporary Clinical Trials|April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophyMichela Guglieri, Kate Bushby, Michael P McDermott, 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|July 3, 2020
Minimal manifestation status and prednisone withdrawal in the MGTX trialIkjae Lee, Hui-Chien Kuo, Inmaculada B Aban, et al.
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