Division of Neuromuscular Medicine

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  • 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, Feb 2022, In: Annals of Neurology. 91, 2, p. 165-175 11 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
  • An online non-meditative mindfulness intervention for people with ALS and their caregivers: a randomized controlled trial

    Pagnini, F., Phillips, D., Haulman, A., Bankert, M., Simmons, Z. & Langer, E., 2022, In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 23, 1-2, p. 116-127 12 p.

    Research output: Contribution to journalArticlepeer-review

    2 Scopus citations
  • Ethical Perspectives on Treatment Options with Spinal Muscular Atrophy Patients

    Yeo, C. J. J., Simmons, Z., De Vivo, D. C. & Darras, B. T., Mar 2022, In: Annals of Neurology. 91, 3, p. 305-316 12 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    1 Scopus citations
  • Laryngospasm: A frequently underrecognized symptom in amyotrophic lateral sclerosis

    Su, X. W. & Simmons, Z., Apr 2022, In: Muscle and Nerve. 65, 4, p. 367-368 2 p.

    Research output: Contribution to journalEditorialpeer-review

  • An integrated multi-omic analysis of iPSC-derived motor neurons from C9ORF72 ALS patients

    The NeuroLINCS Consortium & NYGC ALS Consortium, Nov 19 2021, In: iScience. 24, 11, 103221.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    2 Scopus citations
  • A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS

    Shefner, J. M., Andrews, J. A., Genge, A., Jackson, C., Lechtzin, N., Miller, T. M., Cockroft, B. M., Meng, L., Wei, J., Wolff, A. A., Malik, F. I., Bodkin, C., Brooks, B. R., Caress, J., Dionne, A., Fee, D., Goutman, S. A., Goyal, N. A., Hardiman, O., Hayat, G. & 33 others, Heiman-Patterson, T., Heitzman, D., Henderson, R. D., Johnston, W., Karam, C., Kiernan, M. C., Kolb, S. J., Korngut, L., Ladha, S., Matte, G., Mora, J. S., Needham, M., Oskarsson, B., Pattee, G. L., Pioro, E. P., Pulley, M., Quan, D., Rezania, K., Schellenberg, K. L., Schultz, D., Shoesmith, C., Simmons, Z., Statland, J., Sultan, S., Swenson, A., Berg, L. H. V. D., Vu, T., Vucic, S., Weiss, M., Whyte-Rayson, A., Wymer, J., Zinman, L. & Rudnicki, S. A., 2021, In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 22, 3-4, p. 287-299 13 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    12 Scopus citations
  • Association of Variants in the SPTLC1 Gene with Juvenile Amyotrophic Lateral Sclerosis

    Johnson, J. O., Chia, R., Miller, D. E., Li, R., Kumaran, R., Abramzon, Y., Alahmady, N., Renton, A. E., Topp, S. D., Gibbs, J. R., Cookson, M. R., Sabir, M. S., Dalgard, C. L., Troakes, C., Jones, A. R., Shatunov, A., Iacoangeli, A., Al Khleifat, A., Ticozzi, N., Silani, V. & 274 others, Gellera, C., Blair, I. P., Dobson-Stone, C., Kwok, J. B., Bonkowski, E. S., Palvadeau, R., Tienari, P. J., Morrison, K. E., Shaw, P. J., Al-Chalabi, A., Brown, R. H., Calvo, A., Mora, G., Al-Saif, H., Gotkine, M., Leigh, F., Chang, I. J., Perlman, S. J., Glass, I., Scott, A. I., Shaw, C. E., Basak, A. N., Landers, J. E., Chiò, A., Crawford, T. O., Smith, B. N., Traynor, B. J., Smith, B. N., Ticozzi, N., Fallini, C., Gkazi, A. S., Topp, S. D., Scotter, E. L., Kenna, K. P., Keagle, P., Tiloca, C., Vance, C., Troakes, C., Colombrita, C., King, A., Pensato, V., Castellotti, B., Baas, F., Ten Asbroek, A. L. M. A., McKenna-Yasek, D., McLaughlin, R. L., Polak, M., Asress, S., Esteban-Pérez, J., Stevic, Z., D'Alfonso, S., Mazzini, L., Comi, G. P., Del Bo, R., Ceroni, M., Gagliardi, S., Querin, G., Bertolin, C., Van Rheenen, W., Rademakers, R., Van Blitterswijk, M., Lauria, G., Duga, S., Corti, S., Cereda, C., Corrado, L., Sorarù, G., Williams, K. L., Nicholson, G. A., Blair, I. P., Leblond-Manry, C., Rouleau, G. A., Hardiman, O., Morrison, K. E., Veldink, J. H., Van Den Berg, L. H., Al-Chalabi, A., Pall, H., Shaw, P. J., Turner, M. R., Talbot, K., Taroni, F., García-Redondo, A., Wu, Z., Glass, J. D., Gellera, C., Ratti, A., Brown, R. H., Silani, V., Shaw, C. E., Landers, J. E., Dalgard, C. L., Adeleye, A., Soltis, A. R., Alba, C., Viollet, C., Bacikova, D., Hupalo, D. N., Sukumar, G., Pollard, H. B., Wilkerson, M. D., Martinez, E. M. G., Abramzon, Y., Ahmed, S., Arepalli, S., Baloh, R. H., Bowser, R., Brady, C. B., Brice, A., Broach, J., Campbell, R. H., Camu, W., Cooper-Knock, J., Ding, J., Drepper, C., Drory, V. E., Dunckley, T. L., Eicher, J. D., England, B. K., Faghri, F., Feldman, E., Floeter, M. K., Fratta, P., Geiger, J. T., Gerhard, G., Gibbs, J. R., Gibson, S. B., Glass, J. D., Hardy, J., Harms, M. B., Heiman-Patterson, T. D., Hernandez, D. G., Jansson, L., Kirby, J., Kowall, N. W., Laaksovirta, H., Landeck, N., Landi, F., Le Ber, I., Lumbroso, S., Macgowan, D. J. L., Maragakis, N. J., Mouzat, K., Murphy, N. A., Myllykangas, L., Nalls, M. A., Orrell, R. W., Ostrow, L. W., Pamphlett, R., Pickering-Brown, S., Pioro, E. P., Pletnikova, O., Pliner, H. A., Pulst, S. M., Ravits, J. M., Renton, A. E., Rivera, A., Robberecht, W., Rogaeva, E., Rollinson, S., Rothstein, J. D., Scholz, S. W., Sendtner, M., Shaw, P. J., Sidle, K. C., Simmons, Z., Singleton, A. B., Smith, N., Stone, D. J., Tienari, P. J., Troncoso, J. C., Valori, M., Van Damme, P., Van Deerlin, V. M., Van Den Bosch, L., Zinman, L., Landers, J. E., Chiò, A., Traynor, B. J., Angelocola, S. M., Ausiello, F. P., Barberis, M., Bartolomei, I., Battistini, S., Bersano, E., Bisogni, G., Borghero, G., Brunetti, M., Cabona, C., Calvo, A., Canale, F., Canosa, A., Cantisani, T. A., Capasso, M., Caponnetto, C., Cardinali, P., Carrera, P., Casale, F., Chiò, A., Colletti, T., Conforti, F. L., Conte, A., Conti, E., Corbo, M., Cuccu, S., Dalla Bella, E., D'Errico, E., Demarco, G., Dubbioso, R., Ferrarese, C., Ferraro, P. M., Filippi, M., Fini, N., Floris, G., Fuda, G., Gallone, S., Gianferrari, G., Giannini, F., Grassano, M., Greco, L., Iazzolino, B., Introna, A., La Bella, V., Lattante, S., Liguori, R., Logroscino, G., Logullo, F. O., Lunetta, C., Mandich, P., Mandrioli, J., Manera, U., Manganelli, F., Marangi, G., Marinou, K., Marrosu, M. G., Martinelli, I., Messina, S., Moglia, C., Mosca, L., Murru, M. R., Origone, P., Passaniti, C., Petrelli, C., Petrucci, A., Pozzi, S., Pugliatti, M., Quattrini, A., Ricci, C., Riolo, G., Riva, N., Russo, M., Sabatelli, M., Salamone, P., Salivetto, M., Salvi, F., Santarelli, M., Sbaiz, L., Sideri, R., Simone, I., Simonini, C., Spataro, R., Tanel, R., Tedeschi, G., Ticca, A., Torriello, A., Tranquilli, S., Tremolizzo, L., Trojsi, F., Vasta, R., Vacchiano, V., Vita, G., Volanti, P., Zollino, M. & Zucchi, E., Oct 2021, In: JAMA neurology. 78, 10, p. 1236-1248 13 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    2 Scopus citations
  • Autonomic neuropathies

    Kaur, D., Tiwana, H., Stino, A. & Sandroni, P., Jan 2021, In: Muscle and Nerve. 63, 1, p. 10-21 12 p.

    Research output: Contribution to journalReview articlepeer-review

    9 Scopus citations
  • Coexistence of TDP-43 and C5b-9 staining of muscle in a patient with inclusion body myositis

    Law, C., Li, H. & Bandyopadhyay, S., Feb 9 2021, In: BMJ case reports. 14, 2, e238312.

    Research output: Contribution to journalArticlepeer-review

  • Effect of Ezogabine on Cortical and Spinal Motor Neuron Excitability in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial

    Wainger, B. J., Macklin, E. A., Vucic, S., McIlduff, C. E., Paganoni, S., Maragakis, N. J., Bedlack, R., Goyal, N. A., Rutkove, S. B., Lange, D. J., Rivner, M. H., Goutman, S. A., Ladha, S. S., Mauricio, E. A., Baloh, R. H., Simmons, Z., Pothier, L., Kassis, S. B., La, T., Hall, M. & 29 others, Evora, A., Klements, D., Hurtado, A., Pereira, J. D., Koh, J., Celnik, P. A., Chaudhry, V., Gable, K., Juel, V. C., Phielipp, N., Marei, A., Rosenquist, P., Meehan, S., Oskarsson, B., Lewis, R. A., Kaur, D., Kiskinis, E., Woolf, C. J., Eggan, K., Weiss, M. D., Berry, J. D., David, W. S., Davila-Perez, P., Camprodon, J. A., Pascual-Leone, A., Kiernan, M. C., Shefner, J. M., Atassi, N. & Cudkowicz, M. E., Feb 2021, In: JAMA neurology. 78, 2, p. 186-196 11 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    25 Scopus citations