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Page 1
Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.
Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGAIN Study Group. Howard JF Jr, et al. Lancet Neurol. 2017 Dec;16(12):976-986. doi: 10.1016/S1474-4422(17)30369-1. Epub 2017 Oct 20. Lancet Neurol. 2017. PMID: 29066163 Clinical Trial.
A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease.
McGarry A, McDermott M, Kieburtz K, de Blieck EA, Beal F, Marder K, Ross C, Shoulson I, Gilbert P, Mallonee WM, Guttman M, Wojcieszek J, Kumar R, LeDoux MS, Jenkins M, Rosas HD, Nance M, Biglan K, Como P, Dubinsky RM, Shannon KM, O'Suilleabhain P, Chou K, Walker F, Martin W, Wheelock VL, McCusker E, Jankovic J, Singer C, Sanchez-Ramos J, Scott B, Suchowersky O, Factor SA, Higgins DS Jr, Molho E, Revilla F, Caviness JN, Friedman JH, Perlmutter JS, Feigin A, Anderson K, Rodriguez R, McFarland NR, Margolis RL, Farbman ES, Raymond LA, Suski V, Kostyk S, Colcher A, Seeberger L, Epping E, Esmail S, Diaz N, Fung WL, Diamond A, Frank S, Hanna P, Hermanowicz N, Dure LS, Cudkowicz M; Huntington Study Group 2CARE Investigators and Coordinators. McGarry A, et al. Neurology. 2017 Jan 10;88(2):152-159. doi: 10.1212/WNL.0000000000003478. Epub 2016 Dec 2. Neurology. 2017. PMID: 27913695 Free PMC article. Clinical Trial.
Motor, cognitive, and functional declines contribute to a single progressive factor in early HD.
Schobel SA, Palermo G, Auinger P, Long JD, Ma S, Khwaja OS, Trundell D, Cudkowicz M, Hersch S, Sampaio C, Dorsey ER, Leavitt BR, Kieburtz KD, Sevigny JJ, Langbehn DR, Tabrizi SJ; TRACK-HD, COHORT, CARE-HD, and 2CARE Huntington Study Group Investigators. Schobel SA, et al. Neurology. 2017 Dec 12;89(24):2495-2502. doi: 10.1212/WNL.0000000000004743. Epub 2017 Nov 15. Neurology. 2017. PMID: 29142089 Free PMC article.
Plasma metabolomic biomarker panel to distinguish patients with amyotrophic lateral sclerosis from disease mimics.
Lawton KA, Brown MV, Alexander D, Li Z, Wulff JE, Lawson R, Jaffa M, Milburn MV, Ryals JA, Bowser R, Cudkowicz ME, Berry JD; Northeast ALS Consortium. Lawton KA, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):362-70. doi: 10.3109/21678421.2014.908311. Epub 2014 Jul 1. Amyotroph Lateral Scler Frontotemporal Degener. 2014. PMID: 24984169
A randomized controlled trial of methotrexate for patients with generalized myasthenia gravis.
Pasnoor M, He J, Herbelin L, Burns TM, Nations S, Bril V, Wang AK, Elsheikh BH, Kissel JT, Saperstein D, Shaibani JA, Jackson C, Swenson A, Howard JF Jr, Goyal N, David W, Wicklund M, Pulley M, Becker M, Mozaffar T, Benatar M, Pazcuzzi R, Simpson E, Rosenfeld J, Dimachkie MM, Statland JM, Barohn RJ; Methotrexate in MG Investigators of the Muscle Study Group. Pasnoor M, et al. Neurology. 2016 Jul 5;87(1):57-64. doi: 10.1212/WNL.0000000000002795. Epub 2016 Jun 15. Neurology. 2016. PMID: 27306628 Free PMC article. Clinical Trial.
A multi-center screening trial of rasagiline in patients with amyotrophic lateral sclerosis: Possible mitochondrial biomarker target engagement.
Macchi Z, Wang Y, Moore D, Katz J, Saperstein D, Walk D, Simpson E, Genge A, Bertorini T, Fernandes JA, Swenson A, Elman L, Dimachkie M, Herbelin L, Miller J, Lu J, Wilkins H, Swerdlow RH, Statland J, Barohn R; Western ALS (WALS) Rasagiline Study Group. Macchi Z, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(5-6):345-52. doi: 10.3109/21678421.2015.1026826. Epub 2015 Apr 2. Amyotroph Lateral Scler Frontotemporal Degener. 2015. PMID: 25832828 Free PMC article.
Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS.
Miller RG, Block G, Katz JS, Barohn RJ, Gopalakrishnan V, Cudkowicz M, Zhang JR, McGrath MS, Ludington E, Appel SH, Azhir A; Phase 2 Trial NP001 Investigators. Miller RG, et al. Neurol Neuroimmunol Neuroinflamm. 2015 Apr 9;2(3):e100. doi: 10.1212/NXI.0000000000000100. eCollection 2015 Jun. Neurol Neuroimmunol Neuroinflamm. 2015. PMID: 25884010 Free PMC article.