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Page 1
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.
Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. Miller TM, et al. Among authors: simpson e. Lancet Neurol. 2013 May;12(5):435-42. doi: 10.1016/S1474-4422(13)70061-9. Epub 2013 Mar 29. Lancet Neurol. 2013. PMID: 23541756 Free PMC article. Clinical Trial.
TREM2 variant p.R47H as a risk factor for sporadic amyotrophic lateral sclerosis.
Cady J, Koval ED, Benitez BA, Zaidman C, Jockel-Balsarotti J, Allred P, Baloh RH, Ravits J, Simpson E, Appel SH, Pestronk A, Goate AM, Miller TM, Cruchaga C, Harms MB. Cady J, et al. Among authors: simpson e. JAMA Neurol. 2014 Apr;71(4):449-53. doi: 10.1001/jamaneurol.2013.6237. JAMA Neurol. 2014. PMID: 24535663 Free PMC article.
Safety and efficacy of ceftriaxone for amyotrophic lateral sclerosis: a multi-stage, randomised, double-blind, placebo-controlled trial.
Cudkowicz ME, Titus S, Kearney M, Yu H, Sherman A, Schoenfeld D, Hayden D, Shui A, Brooks B, Conwit R, Felsenstein D, Greenblatt DJ, Keroack M, Kissel JT, Miller R, Rosenfeld J, Rothstein JD, Simpson E, Tolkoff-Rubin N, Zinman L, Shefner JM; Ceftriaxone Study Investigators. Cudkowicz ME, et al. Among authors: simpson e. Lancet Neurol. 2014 Nov;13(11):1083-1091. doi: 10.1016/S1474-4422(14)70222-4. Epub 2014 Oct 5. Lancet Neurol. 2014. PMID: 25297012 Free PMC article. Clinical Trial.
Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial.
Aggarwal SP, Zinman L, Simpson E, McKinley J, Jackson KE, Pinto H, Kaufman P, Conwit RA, Schoenfeld D, Shefner J, Cudkowicz M; Northeast and Canadian Amyotrophic Lateral Sclerosis consortia. Aggarwal SP, et al. Among authors: simpson e. Lancet Neurol. 2010 May;9(5):481-8. doi: 10.1016/S1474-4422(10)70068-5. Epub 2010 Apr 1. Lancet Neurol. 2010. PMID: 20363190 Free PMC article. Clinical Trial.
Toward more efficient clinical trials for amyotrophic lateral sclerosis.
Cudkowicz ME, Katz J, Moore DH, O'Neill G, Glass JD, Mitsumoto H, Appel S, Ravina B, Kieburtz K, Shoulson I, Kaufmann P, Khan J, Simpson E, Shefner J, Levin B, Cwik V, Schoenfeld D, Aggarwal S, McDermott MP, Miller RG. Cudkowicz ME, et al. Among authors: simpson e. Amyotroph Lateral Scler. 2010 May 3;11(3):259-65. doi: 10.3109/17482960903358865. Amyotroph Lateral Scler. 2010. PMID: 19961263 Review.
Design and initial results of a multi-phase randomized trial of ceftriaxone in amyotrophic lateral sclerosis.
Berry JD, Shefner JM, Conwit R, Schoenfeld D, Keroack M, Felsenstein D, Krivickas L, David WS, Vriesendorp F, Pestronk A, Caress JB, Katz J, Simpson E, Rosenfeld J, Pascuzzi R, Glass J, Rezania K, Rothstein JD, Greenblatt DJ, Cudkowicz ME; Northeast ALS Consortium. Berry JD, et al. Among authors: simpson e. PLoS One. 2013 Apr 17;8(4):e61177. doi: 10.1371/journal.pone.0061177. Print 2013. PLoS One. 2013. PMID: 23613806 Free PMC article. Clinical Trial.
Systemic pharmacokinetics and cerebrospinal fluid uptake of intravenous ceftriaxone in patients with amyotrophic lateral sclerosis.
Zhao Y, Cudkowicz ME, Shefner JM, Krivickas L, David WS, Vriesendorp F, Pestronk A, Caress JB, Katz J, Simpson E, Rosenfeld J, Pascuzzi R, Glass J, Rezania K, Harmatz JS, Schoenfeld D, Greenblatt DJ. Zhao Y, et al. Among authors: simpson e. J Clin Pharmacol. 2014 Oct;54(10):1180-7. doi: 10.1002/jcph.317. Epub 2014 May 16. J Clin Pharmacol. 2014. PMID: 24771634 Free PMC article. Clinical Trial.
Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability.
Berry JD, Paganoni S, Atassi N, Macklin EA, Goyal N, Rivner M, Simpson E, Appel S, Grasso DL, Mejia NI, Mateen F, Gill A, Vieira F, Tassinari V, Perrin S. Berry JD, et al. Among authors: simpson e. Muscle Nerve. 2017 Dec;56(6):1077-1084. doi: 10.1002/mus.25733. Epub 2017 Aug 29. Muscle Nerve. 2017. PMID: 28662296 Free PMC article. Clinical Trial.
Genome-wide Analyses Identify KIF5A as a Novel ALS Gene.
Nicolas A, Kenna KP, Renton AE, Ticozzi N, Faghri F, Chia R, Dominov JA, Kenna BJ, Nalls MA, Keagle P, Rivera AM, van Rheenen W, Murphy NA, van Vugt JJFA, Geiger JT, Van der Spek RA, Pliner HA, Shankaracharya, Smith BN, Marangi G, Topp SD, Abramzon Y, Gkazi AS, Eicher JD, Kenna A; ITALSGEN Consortium; Mora G, Calvo A, Mazzini L, Riva N, Mandrioli J, Caponnetto C, Battistini S, Volanti P, La Bella V, Conforti FL, Borghero G, Messina S, Simone IL, Trojsi F, Salvi F, Logullo FO, D'Alfonso S, Corrado L, Capasso M, Ferrucci L; Genomic Translation for ALS Care (GTAC) Consortium; Moreno CAM, Kamalakaran S, Goldstein DB; ALS Sequencing Consortium; Gitler AD, Harris T, Myers RM; NYGC ALS Consortium; Phatnani H, Musunuri RL, Evani US, Abhyankar A, Zody MC; Answer ALS Foundation; Kaye J, Finkbeiner S, Wyman SK, LeNail A, Lima L, Fraenkel E, Svendsen CN, Thompson LM, Van Eyk JE, Berry JD, Miller TM, Kolb SJ, Cudkowicz M, Baxi E; Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium; Benatar M, Taylor JP, Rampersaud E, Wu G, Wuu J; SLAGEN Consortium; Lauria G, Verde F, Fogh I, Tiloca C, Comi GP, Sorarù G, Cereda C; French ALS Consortium; Corcia P, Laaks… See abstract for full author list ➔ Nicolas A, et al. Neuron. 2018 Mar 21;97(6):1267-1288. doi: 10.1016/j.neuron.2018.02.027. Neuron. 2018. PMID: 29566793 Free PMC article.
Seeking a measure of clinically meaningful change in ALS.
McElhiney M, Rabkin JG, Goetz R, Katz J, Miller RG, Forshew DA, David W, Cudkowicz M, Glass JD, Appel S, Simpson E, Mitsumoto H. McElhiney M, et al. Among authors: simpson e. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):398-405. doi: 10.3109/21678421.2014.942668. Epub 2014 Aug 15. Amyotroph Lateral Scler Frontotemporal Degener. 2014. PMID: 25124889
2,060 results