Simpson lab Publications


Neurogenic Orthostatic Hypotension: An Iatrogenic Complication of Successful Pancreas Transplantation?
Kuten, SA, Simpson, EP, Gaber, AO, Nguyen, DTM, Graviss, EA, Patel, S & Knight, RJ 2017, .

Improving symptom management for people with amyotrophic lateral sclerosis
Nicholson, K, Murphy, A, Mcdonnell, E, Shapiro, J, Simpson, E, Glass, J, Mitsumoto, H, Forshew, D, Miller, R & Atassi, N 2017, Muscle and Nerve. DOI:

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, 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 2016, Neurology, vol 87, no. 1, pp. 57-64. DOI:

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 2015, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 16, no. 5-6, pp. 345-352. DOI:

Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways
Cirulli, ET, Lasseigne, BN, Petrovski, S, Sapp, PC, Dion, PA, Leblond, CS, Couthouis, J, Lu, Y-F, Wang, Q, Krueger, BJ, Ren, Z, Keebler, J, Han, Y, Levy, SE, Boone, BE, Wimbish, JR, Waite, LL, Jones, AL, Carulli, JP, Day-Williams, AG, Staropoli, JF, Xin, WW, Chesi, A, Raphael, AR, McKenna-Yasek, D, Cady, J, Vianney de Jong, JMB, Kenna, KP, Smith, BN, Topp, S, Miller, J, Gkazi, A, Al-Chalabi, A, van den Berg, LH, Veldink, J, Silani, V, Ticozzi, N, Shaw, CE, Baloh, RH, Appel, S, Simpson, E, Lagier-Tourenne, C, Pulst, SM, Gibson, S, Trojanowski, JQ, Elman, L, McCluskey, L, Grossman, M, Shneider, NA, Chung, WK, Ravits, JM, Glass, JD, Sims, KB, Van Deerlin, VM, Maniatis, T, Hayes, SD, Ordureau, A, Swarup, S, Landers, J, Baas, F, Allen, AS, Bedlack, RS, Harper, JW, Gitler, AD, Rouleau, GA, Brown, R, Harms, MB, Cooper, GM, Harris, T, Myers, RM, Goldstein, DB & FALS Sequencing Consortium 2015, Science (New York, N.Y.), vol 347, no. 6229, pp. 1436-41. DOI:

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 2014, Journal of Clinical Pharmacology, vol 54, no. 10, pp. 1180-1187. DOI:

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, Kalra, S, Korngut, L, Omar-Crawford, H, Sekhon, R, White, C, Benstead, T, Grant, I, Reidy, S, McIntosh, C, McKinley, J, Shoesmith, C, Zinman, L, Botez, S, Bouchard, JP, DAmour, M, Genge, A, Hank, N, Levine, T, Saperstein, D, Alvarez, R, Banda, C, Garcia, R, Graves, M, Gruendler, H, Katz, J, Lin, F, Lomen-Hoerth, C, Martin, V, Miller, R, Moses, D, Mozaffar, T, Nist, L, Oskarsson, B, Rosenfeld, J, Tsimerinov, E, Tully, P, Villierme, C, Voelz, K, Wiedau-Pazos, M, Rollins, Y, Felice, K, Bayat, E, Kelly, AN, Boylan, KB, DeSaro, P, Koggan, D, Verma, A, Bordeau, J, Glass, J, Polak, M, Quarles, B, Rivner, MH, Casey, P, Sufit, R, Bodkin, C, Guingrich, S, Kincaid, J, Micheels, A, Pascuzzi, R, Snook, R, Barohn, RJ, Dick, A, Herbelin, L, Dimachkie, MM, McVey, AL, Walsh, M, Wang, Y, Hutchison, J, Kasarskis, E, King, J, Tandy, T, Thomas, S, Vanderpool, K, Rothstein, J, Andres, P, Bellanich, M, Cudkowicz, ME, David, W, Goldenberg, A, Greenblatt, DJ, Krivickas, L, Loci, L, Majkut, M, OConnor, O, Parkinson, M, Pulley, D, Russell, J, Sullivan, L, Foley, H, Gelinas, D, Newman, DS, Bundlie, S, Leviton, T, Patel, S, Rohde, C, Swanson, S, Tiryaki, E, Fann, A, Hayat, G, Pestronk, A, Pattee, GL, Weber, B, Keroack, M, Belsh, B, Belsh, J, Mertz, MA, DeNero, AM, Imperato, T, Lange, DJ, Kassebaum, N, MacGowan, D, Mitsumoto, H, Scelsa, SN, Shahbazi, M, Shefner, JM, Simionescu, L, Watson, ML, Wymer, J, Bravver, E, Brooks, BR, Caress, J, Cartwright, MS, Johnston-Crews, T, Lary, C, Shuh, J, Bartlett, A, Berry, N, Kissel, JT, Kuenzler, R, Pioro, EP, Quick, A, Ash, B, Goslin, K, Deboo, A, Giampole, A, Heiman-Patterson, T, Lacomis, D, McCluskey, L, Powell, M, Rana, S, Rojas, L, Rowlands, D, Simmons, Z, Stephens, HE, DeCandio, M, Stickler, D, Davis, DL, Donofrio, PD, Halton, S, Hand, S, Hastings, M, Heitzman, D, Lay, L, McCloskey, J, Simpson, E, Bromberg, M, Joshi, A & Phillips, LH 2014, The Lancet Neurology, vol 13, no. 11, pp. 1083-1091. DOI:

Correction to: '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 2014, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 15, no. 7-8. DOI:

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 2014, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 15, no. 5-6, pp. 398-405. DOI:

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 2014, JAMA Neurology, vol 71, no. 4, pp. 449-453. DOI:

Hypercaloric enteral nutrition in patients with amyotrophic lateral sclerosis: A randomised, double-blind, placebo-controlled phase 2 trial
Wills, AM, Hubbard, J, Macklin, EA, Glass, J, Tandan, R, Simpson, EP, Brooks, B, Gelinas, D, Mitsumoto, H, Mozaffar, T, Hanes, GP, Ladha, SS, Heiman-Patterson, T, Katz, J, Lou, JS, Mahoney, K, Grasso, D, Lawson, R, Yu, H & Cudkowicz, M 2014, The Lancet, vol 383, no. 9934, pp. 2065-2072. DOI:

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 2013, The Lancet Neurology, vol 12, no. 5, pp. 435-442. DOI:

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 2013, PLoS ONE, vol 8, no. 4, e61177. DOI:

Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: Examining a more efficient trial design
Miller, RG, Moore, DH, Forshew, DA, Katz, JS, Barohn, RJ, Valan, M, Bromberg, MB, Goslin, KL, Graves, MC, McCluskey, LF, McVey, AL, Mozaffar, T, Florence, JM, Pestronk, A, Ross, M, Simpson, EP & Appel, SH 2011, Neurology, vol 77, no. 10, pp. 973-979. DOI:

Distinctive genetic and clinical features of CMT4J: A severe neuropathy caused by mutations in the PI(3,5)P2 phosphatase FIG4
Nicholson, G, Lenk, GM, Reddel, SW, Grant, AE, Towne, CF, Ferguson, CJ, Simpson, E, Scheuerle, A, Yasick, M, Hoffman, S, Blouin, R, Brandt, C, Coppola, G, Biesecker, LG, Batish, SD & Meisler, MH 2011, Brain, vol 134, no. 7, pp. 1959-1971. DOI:

Does apolipoprotein E genotype modify the clinical expression of ALS?
Jawaid, A, Poon, M, Strutt, AM, Rice, LK, Mcdowell, EJ, Salamone, AR, Qureshi, SU, Simpson, E, Appel, SH, York, MK & Schulz, PE 2011, European Journal of Neurology, vol 18, no. 4, pp. 618-624. DOI:

A decrease in body mass index is associated with faster progression of motor symptoms and shorter survival in ALS
Jawaid, A, Murthy, SB, Wilson, AM, Qureshi, SU, Amro, MJ, Wheaton, M, Simpson, E, Harati, Y, Strutt, AM, York, MK & Schulz, PE 2010, Amyotrophic Lateral Sclerosis, vol 11, no. 6, pp. 542-548. DOI:

Myelopathies
Wright, R & Simpson, EP 2010, . in Neurology Secrets. Elsevier, pp. 131-140. DOI:

Neurologic Complications of Systemic Disease
Simpson, EP 2010, . in Neurology Secrets. Elsevier, pp. 353-373. DOI:

Toward more efficient clinical trials for amyotrophic lateral sclerosis
Cudkowicz, ME, Katz, J, Moore, DH, ONeill, 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 2010, Amyotrophic Lateral Sclerosis, vol 11, no. 3, pp. 259-265. DOI: