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Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1): Protocol of an international natural history study.

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第一作者: Karlien,Mul
第一单位: Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands.
作者单位: Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands. [1] Department of Neurology, University of Rochester Medical Center, Rochester, New York, United States of America. [2] Department of Neurology, Virginia Commonwealth University, Richmond, Virginia, United States of America. [3] Neurorehabilitation Unit, The NeMO Clinical Center, University of Milan, Milan, Italy. [4] Department of Neurology, Université de Sherbrooke, CIUSSS Saguenay-Lac-Saint-Jean, Québec, Canada. [5] Department of Neurology, University of Florida College of Medicine, Gainesville, Florida, United States of America. [6] Centre for Brain Research Neurogenetics Clinic, The University of Auckland, Auckland, New Zealand. [7] Department of Neurology, Division of Neuromuscular Disease, and Department of Pathology and Laboratory Medicine, University of Rochester Medical Center, Rochester, New York, United States of America. [8] Department of Neurology, University of Kansas Medical Center, Kansas City, Kansas, United States of America. [9] Department of Neurology, Division of Neuromuscular Diseases, The Ohio State University Wexner Medical Center, Columbus, Ohio, United States of America. [10] Department of Neurology, The Institute of Neurology, University College London and National Hospital for Neurology and Neurosurgery, Queen Square, London, United Kingdom. [11] Department of Neurology and Neurological Sciences, Stanford University, Stanford, California, United States of America. [12] Department of Neurology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, United States of America. [13] Atkinson-Morley Neuromuscular Centre, St George's University Hospitals NHS Foundation Trust, London, United Kingdom. [14] Department of Neurology, Friedrich-Baur-Institute, LMU Clinic, Munich, Germany. [15] Department of Neurology, University of Iowa, Iowa City, Iowa, United States of America. [16] Department of Neurosciences, University of California, San Diego, California, United States of America. [17] Department of Neurology, University of California, Los Angeles, California, United States of America. [18] Houston Methodist Neurological Institute, Houston, Texas, United States of America. [19] Department of Clinical Laboratory and Biomedical Sciences, Division of Clinical Neurophysiology, Osaka University Graduate School of Medicine, Osaka, Japan. [20] Department of Neurology, UT Health San Antonio, San Antonio, Texas, United States of America. [21]
DOI 10.1371/journal.pone.0331163
PMID 41379803
发布时间 2026-01-08
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