Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.

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第一作者： Samantha,LoRusso

第一单位： Department of Neurology, Ohio State University Wexner Medical Center, 395 W. 12th Ave., 7th Floor, Columbus, OH, 43210, USA.

作者： Samantha,LoRusso ^[1] ; Nicholas E,Johnson ^[2] ; Michael P,McDermott ^[3] ; Katy,Eichinger ^[4] ; Russell J,Butterfield ^[5] ; Elena,Carraro ^[6] ; Kiley,Higgs ^[7] ; Leann,Lewis ^[4] ; Karlien,Mul ^[8] ; Sabrina,Sacconi ^[9] ; Valeria A,Sansone ^[6] ; Perry,Shieh ^[10] ; Baziel,van Engelen ^[8] ; Kathryn,Wagner ^[11] ; Leo,Wang ^[12] ; Jeffrey M,Statland ^[13] ; Rabi,Tawil ^[4] ; ReSolve Investigators and the FSHD CTRN18

作者单位： Department of Neurology, Ohio State University Wexner Medical Center, 395 W. 12th Ave., 7th Floor, Columbus, OH, 43210, USA. ^[1] Department of Neurology, Virginia Commonwealth University, 1101 East Marshall St, PO Box 980599, Richmond, VA, 23298, USA. ^[2] Department of Biostatistics and Computational Biology and Department of Neurology, University of Rochester Medical Center, 265 Crittenden Blvd., CU 420630, Rochester, NY, 14642, USA. ^[3] Department of Neurology, University of Rochester Medical Center, Box 673, 601 Elmwood Ave, Rochester, NY, 14642, USA. ^[4] Department of Pediatrics and Neurology, University of Utah, Eccles Institute of Human Genetics, Room 2260A, 15 N 2030 E, Salt Lake City, UT, 84112, USA. ^[5] The NEMO Clinical Center, Neurorehabilitation Unit, University of Milan, Piazza dell'Ospedale Maggiore, 3, Milan, 20162, Italy. ^[6] Department of Neurology, University of Kansas Medical Center, 3901 Rainbow Blvd, MS 2012, Kansas City, KS, 66160, USA. ^[7] Department of Neurology, Radboud University Medical Center, Reinier Postlaan 4 (935), 6525, GC, Nijmegen, The Netherlands. ^[8] Université Côte d'Azur, Peripheral Nervous System, Centre Hospitalier Universitaire de Nice, Muscle & ALS Department, Pasteur 2 Hospital, 30 Voie Romaine, 06001, Nice Cedex 1, France. ^[9] Department of Neurology, University of California, Los Angeles, 300 Medical Plaza, Suite B-200, Los Angeles, CA, 90095, USA. ^[10] Center for Genetic Muscle Disorders, Kennedy Krieger Institute, 707 N. Broadway, Baltimore, MD, USA. ^[11] Department of Neurology, University of Washington, 1959 NE Pacific St, Seattle, WA, 98195, USA. ^[12] Department of Neurology, University of Kansas Medical Center, 3901 Rainbow Blvd, MS 2012, Kansas City, KS, 66160, USA. jstatland@kumc.edu. ^[13]

关键词 Biomarkers Clinical trial Electrical impedance Myography Facioscapulohumeral muscular dystrophy Functional testing Muscular dystrophy Outcome measures

主题词疾病恶化(Disease Progression);肌电描记术(Electromyography);人类(Humans);纵向研究(Longitudinal Studies);肌营养不良, 面肩肱型(Muscular Dystrophy, Facioscapulohumeral);前瞻性研究(Prospective Studies);结果可重复性(Reproducibility of Results)

DOI 10.1186/s12883-019-1452-x

PMID 31506080

发布时间 2020-04-02

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