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Page 1
Seven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials.
Cudkowicz M, Chase MK, Coffey CS, Ecklund DJ, Thornell BJ, Lungu C, Mahoney K, Gutmann L, Shefner JM, Staley KJ, Bosch M, Foster E, Long JD, Bayman EO, Torner J, Yankey J, Peters R, Huff T, Conwit RA; NeuroNEXT Clinical Study Sites; Shinnar S, Patch D, Darras BT, Ellis A, Packer RJ, Marder KS, Chiriboga CA, Henchcliffe C, Moran JA, Nikolov B, Factor SA, Seeley C, Greenberg SM, Amato AA, DeGregorio S, Simuni T, Ward T, Kissel JT, Kolb SJ, Bartlett A, Quinn JF, Keith K, Levine SR, Gilles N, Coyle PK, Lamb J, Wolfe GI, Crumlish A, Mejico L, Iqbal MM, Bowen JD, Tongco C, Nabors LB, Bashir K, Benge M, McDonald CM, Henricson EK, Oskarsson B, Dobkin BH, Canamar C, Glauser TA, Woo D, Molloy A, Clark P, Vollmer TL, Stein AJ, Barohn RJ, Dimachkie MM, Le Pichon JB, Benatar MG, Steele J, Wechsler L, Clemens PR, Amity C, Holloway RG, Annis C, Goldberg MP, Andersen M, Iannaccone ST, Smith AG, Singleton JR, Doudova M, Haley EC, Quigg MS, Lowenhaupt S, Malow BA, Adkins K, Clifford DB, Teshome MA, Connolly N. Cudkowicz M, et al. Among authors: mcdonald cm. JAMA Neurol. 2020 Jun 1;77(6):755-763. doi: 10.1001/jamaneurol.2020.0367. JAMA Neurol. 2020. PMID: 32202612 Free PMC article.
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group; Spiegel R, Barth J, Elfring G, Reha A, Peltz SW. McDonald CM, et al. Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17. Muscle Nerve. 2013. PMID: 23674289 Free PMC article. Clinical Trial.
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.
McDonald CM, Henricson EK, Abresch RT, Florence JM, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group; Spiegel R, Barth J, Elfring G, Reha A, Peltz S. McDonald CM, et al. Muscle Nerve. 2013 Sep;48(3):343-56. doi: 10.1002/mus.23902. Epub 2013 Jun 26. Muscle Nerve. 2013. PMID: 23681930 Free PMC article. Clinical Trial.
The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year.
Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, de Bie E, McDonald CM. Henricson E, et al. Among authors: mcdonald cm. PLoS Curr. 2013 Jul 8;5:ecurrents.md.9e17658b007eb79fcd6f723089f79e06. doi: 10.1371/currents.md.9e17658b007eb79fcd6f723089f79e06. PLoS Curr. 2013. PMID: 23867975 Free PMC article.
Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study.
Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group Investigators. Bello L, et al. Among authors: mcdonald cm. Ann Neurol. 2015 Apr;77(4):684-96. doi: 10.1002/ana.24370. Epub 2015 Mar 13. Ann Neurol. 2015. PMID: 25641372 Free PMC article.
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
Crow RA, Hart KA, McDermott MP, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Hirtz D, Lochmuller H, Straub V, Ciafaloni E, Shieh PB, Spinty S, Childs AM, Manzur AY, Morandi L, Butterfield RJ, Horrocks I, Roper H, Flanigan KM, Kuntz NL, Mah JK, Morrison L, Darras BT, von der Hagen M, Schara U, Wilichowski E, Mongini T, McDonald CM, Vita G, Barohn RJ, Finkel RS, Wicklund M, McMillan HJ Jr, Hughes I, Pegoraro E, Bryan Burnette W, Howard JF, Thangarajh M, Campbell C, Griggs RC, Bushby K, Guglieri M. Crow RA, et al. Among authors: mcdonald cm. Trials. 2018 May 10;19(1):291. doi: 10.1186/s13063-018-2645-0. Trials. 2018. PMID: 29793540 Free PMC article. Review.
Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.
McDonald CM, Wong B, Flanigan KM, Wilson R, de Kimpe S, Lourbakos A, Lin Z, Campion G; DEMAND V study group. McDonald CM, et al. Ann Clin Transl Neurol. 2018 Jul 11;5(8):913-926. doi: 10.1002/acn3.579. eCollection 2018 Aug. Ann Clin Transl Neurol. 2018. PMID: 30128316 Free PMC article.
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