Simpson lab Publications


Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Thornton, CA, Moxley, RT, Eichinger, K, Heatwole, C, Mignon, L, Arnold, WD, Ashizawa, T, Day, JW, Dent, G, Tanner, MK, Duong, T, Greene, EP, Herbelin, L, Johnson, NE, King, W, Kissel, JT, Leung, DG, Lott, DJ, Norris, DA, Pucillo, EM, Schell, W, Statland, JM, Stinson, N, Subramony, SH, Xia, S, Bishop, KM & Bennett, CF 2023, , The Lancet Neurology, vol. 22, no. 3, pp. 218-228. https://doi.org/10.1016/S1474-4422(23)00001-7

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
for the Healey ALS Platform Trial Study Group, HEALEY ALS Platform Trial Study Group, Biostatisticians, Investigators, Clinical Operations Team & Medical Monitors 2022, , Annals of Neurology, vol. 91, no. 2, pp. 165-175. https://doi.org/10.1002/ana.26285


Neurogenic orthostatic hypotension: A common complication of successful pancreas transplantation
Kuten, SA, Graviss, EA, Nguyen, DT, Gaber, AO, Sadhu, AR, Simpson, EP, Yi, SG, Podder, H, Kagan, A & Knight, RJ 2021, , Transplantation Direct, vol. 7, no. 12, e795, pp. e795. https://doi.org/10.1097/TXD.0000000000001208

Decisions With Patients and Families Regarding Aducanumab in Alzheimer Disease, With Recommendations for Consent: AAN Position Statement
the Ethics, Law, and Humanities Committee (a joint committee of the AAN, ANA, and CNS) 2022, , Neurology, vol. 98, no. 4, pp. 154-159. https://doi.org/10.1212/WNL.0000000000013053

Contemporary Neuroscience Core Curriculum for Medical Schools
The AAN Undergraduate Education Subcommittee (UES) & The AAN Education Committee 2021, , Neurology, vol. 97, no. 14, pp. 675-684. https://doi.org/10.1212/WNL.0000000000012664

Fast progression in amyotrophic lateral sclerosis is associated with greater tdp-43 burden in spinal cord
Cathcart, SJ, Appel, SH, Peterson, LE, Greene, EP, Powell, SZ, Arumanayagam, AS, Rivera, AL & Cykowski, MD 2021, , Journal of Neuropathology and Experimental Neurology, vol. 80, no. 8, pp. 754-763. https://doi.org/10.1093/jnen/nlab061

Spinal cord and motor neuron TDP-43 pathology in a sporadic inclusion body myositis patient
Cathcart, SJ, Greene, EP, Powell, SZ, Arumanayagam, AS, Rivera, AL, Tawil, R, Appel, SH & Cykowski, MD 2021, , Journal of Neuropathology and Experimental Neurology, vol. 79, no. 10, pp. 1130-1133. https://doi.org/10.1093/JNEN/NLAA066

Multifocal Noninvasive Magnetic Stimulation of the Primary Motor Cortex in Type 1 Myotonic Dystrophy -A Proof of Concept Pilot Study
Greene, E, Thonhoff, J, John, BS, Rosenfield, DB & Helekar, SA 2021, , Journal of neuromuscular diseases, vol. 8, no. 6, pp. 963-972. https://doi.org/10.3233/JND-210690

Placebo effect in chronic inflammatory demyelinating polyneuropathy: The PATH study and a systematic review
Lewis, RA, Cornblath, DR, Hartung, HP, Sobue, G, Lawo, JP, Mielke, O, Durn, BL, Bril, V, Merkies, ISJ, Bassett, P, Cleasby, A, van Schaik, IN, Sabet, A, George, K, Roberts, L, Carne, R, Blum, S, Henderson, R, Van Damme, P, Demeestere, J, Larue, S, D’Amour, C, Kunc, P, Valis, M, Sussova, J, Kalous, T, Talab, R, Bednar, M, Toomsoo, T, Rubanovits, I, Gross-Paju, K, Sorro, U, Saarela, M, Auranen, M, Pouget, J, Attarian, S, Le Masson, G, Wielanek, A, Desnuelle, C, Delmon, E, Clavelou, P, Aufauvre, D, Schmidt, J, Zschuentzsch, J, Sommer, C, Kramer, D, Hoffmann, O, Goerlitz, C, Haas, J, Chatzopoulos, M, Yoon, R, Gold, R, Berlit, P, Jaspert-Grehl, A, Liebetanz, D, Kutschenko, A, Stangel, M, Trebst, C, Baum, P, Bergh, F, Klehmet, J, Meisel, A, Klostermann, F, Oechtering, J, Lehmann, H, Schroeter, M, Hagenacker, T, Mueller, D, Sperfeld, A, Bethke, F, Drory, IV, Algom, A, Yarnitsky, D, Murinson, B, Di Muzio, A, Ciccocioppo, F, Sorbi, S, Mata, S, Schenone, A, Grandis, M, Lauria, G, Cazzato, D, Antonini, G, Morino, S, Cocito, D, Zibetti, M, Yokota, T, Ohkubo, T, Kanda, T, Kawai, M, Kaida, K, Onoue, H, Kuwabara, S, Mori, M, Iijima, M, Ohyama, K, Baba, M, Tomiyama, M, Nishiyama, K, Akutsu, T, Yokoyama, K, Kanai, K, van Schaik, IN, Eftimov, F, Notermans, NC, Visser, N, Faber, C, Hoeijmakers, J, Rejdak, K, Chyrchel-Paszkiewicz, U, Casanovas Pons, C, Antonia, M, Gamez, J, Salvado, M, Infante, CM, Benitez, S, Lunn, M, Morrow, J, Gosal, D, Lavin, T, Melamed, I, Testori, A, Ajroud-Driss, S, Menichella, D, Simpson, E, Lai, ECH, Dimachkie, M, Barohn, RJ, Beydoun, S, Johl, H, Lange, D, Shtilbans, A, Muley, S, Ladha, S, Freimer, M, Kissel, J, Latov, N, Chin, R, Ubogu, E, Mumfrey, S, Rao, T, MacDonald, P, Sharma, K, Gonzalez, G, Allen, J, Walk, D, Hobson-Webb, L & Gable, K 2020, , Journal of the Peripheral Nervous System, vol. 25, no. 3, pp. 230-237. https://doi.org/10.1111/jns.12402

Immune-Mediated Neuropathies
Fadia, M, Shroff, S & Simpson, E 2019, , Current Treatment Options in Neurology, vol. 21, no. 6, 28. https://doi.org/10.1007/s11940-019-0569-y

Efficacy and safety of IVIG in CIDP: Combined data of the PRIMA and PATH studies
on Behalf of the PRIMA Trial Investigators and the PATH Study Group 2019, , Journal of the Peripheral Nervous System, vol. 24, no. 1, pp. 48-55. https://doi.org/10.1111/jns.12302


Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial
The Rasagiline Investigators of the Muscle Study Group and Western ALS Consortium 2019, , Muscle and Nerve, vol. 59, no. 2, pp. 201-207. https://doi.org/10.1002/mus.26335

Consensus-based care recommendations for adults with myotonic dystrophy type 1
Ashizawa, T, Gagnon, C, Groh, WJ, Gutmann, L, Johnson, NE, Meola, G, Moxley, R, Pandya, S, Rogers, MT, Simpson, E, Angeard, N, Bassez, G, Berggren, KN, Bhakta, D, Bozzali, M, Broderick, A, Byrne, JLB, Campbell, C, Cup, E, Day, JW, De Mattia, E, Duboc, D, Duong, T, Eichinger, K, Ekstrom, AB, Van Engelen, B, Esparis, B, Eymard, B, Ferschl, M, Gadalla, SM, Gallais, B, Goodglick, T, Heatwole, C, Hilbert, J, Holland, V, Kierkegaard, M, Koopman, WJ, Lane, K, Maas, D, Mankodi, A, Mathews, KD, Monckton, DG, Moser, D, Nazarian, S, Nguyen, L, Nopoulos, P, Petty, R, Phetteplace, J, Puymirat, J, Raman, S, Richer, L, Roma, E, Sampson, J, Sansone, V, Schoser, B, Sterling, L, Statland, J, Subramony, SH, Tian, C, Trujillo, C, Tomaselli, G, Turner, C, Verma, A, White, M & Winblad, S 2018, , Neurology: Clinical Practice, vol. 8, no. 6, pp. 507-520. https://doi.org/10.1212/CPJ.0000000000000531

Neuroinflammatory mechanisms in amyotrophic lateral sclerosis pathogenesis
Thonhoff, JR, Simpson, EP & Appel, SH 2018, , Current opinion in neurology, vol. 31, no. 5, pp. 635-639. https://doi.org/10.1097/WCO.0000000000000599

Expanded autologous regulatory T-lymphocyte infusions in ALS A phase I, first-in-human study
Thonhoff, JR, Beers, DR, Zhao, W, Pleitez, M, Simpson, EP, Berry, JD, Cudkowicz, ME & Appel, SH 2018, , Neurology: Neuroimmunology and NeuroInflammation, vol. 5, no. 4, e465, pp. e465. https://doi.org/10.1212/NXI.0000000000000465

Expanded autologous regulatory T-lymphocyte infusions in ALS
Thonhoff, JR, Beers, DR, Zhao, W, Pleitez, M, Simpson, EP, Berry, JD, Cudkowicz, ME & Appel, SH 2018, , Neurology - Neuroimmunology Neuroinflammation, vol. 5, no. 4. <http://nn.neurology.org/content/5/4/e465.abstract>

Intravenous versus subcutaneous immunoglobulin – Authors' reply
PATH study group 2018, , The Lancet Neurology, vol. 17, no. 5, pp. 393-394. https://doi.org/10.1016/S1474-4422(18)30109-1

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene
ITALSGEN Consortium, Genomic Translation for ALS Care (GTAC) Consortium, ALS Sequencing Consortium, NYGC ALS Consortium, Answer ALS Foundation, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, SLAGEN Consortium, French ALS Consortium & Project MinE ALS Sequencing Consortium 2018, , Neuron, vol. 97, no. 6, pp. 1268-1283.e6. https://doi.org/10.1016/j.neuron.2018.02.027