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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
    8 Scopus citations
  • Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines

    Baxi, E. G., Thompson, T., Li, J., Kaye, J. A., Lim, R. G., Wu, J., Ramamoorthy, D., Lima, L., Vaibhav, V., Matlock, A., Frank, A., Coyne, A. N., Landin, B., Ornelas, L., Mosmiller, E., Thrower, S., Farr, S. M., Panther, L., Gomez, E., Galvez, E., & 81 othersPerez, D., Meepe, I., Lei, S., Mandefro, B., Trost, H., Pinedo, L., Banuelos, M. G., Liu, C., Moran, R., Garcia, V., Workman, M., Ho, R., Wyman, S., Roggenbuck, J., Harms, M. B., Stocksdale, J., Miramontes, R., Wang, K., Venkatraman, V., Holewenski, R., Sundararaman, N., Pandey, R., Manalo, D. M., Donde, A., Huynh, N., Adam, M., Wassie, B. T., Vertudes, E., Amirani, N., Raja, K., Thomas, R., Hayes, L., Lenail, A., Cerezo, A., Luppino, S., Farrar, A., Pothier, L., Prina, C., Morgan, T., Jamil, A., Heintzman, S., Jockel-Balsarotti, J., Karanja, E., Markway, J., McCallum, M., Joslin, B., Alibazoglu, D., Kolb, S., Ajroud-Driss, S., Baloh, R., Heitzman, D., Miller, T., Glass, J. D., Patel-Murray, N. L., Yu, H., Sinani, E., Vigneswaran, P., Sherman, A. V., Ahmad, O., Roy, P., Beavers, J. C., Zeiler, S., Krakauer, J. W., Agurto, C., Cecchi, G., Bellard, M., Raghav, Y., Sachs, K., Ehrenberger, T., Bruce, E., Cudkowicz, M. E., Maragakis, N., Norel, R., Van Eyk, J. E., Finkbeiner, S., Berry, J., Sareen, D., Thompson, L. M., Fraenkel, E., Svendsen, C. N. & Rothstein, J. D., Feb 2022, In: Nature neuroscience. 25, 2, p. 226-237 12 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    11 Scopus citations
  • Antisense Oligonucleotides for the Study and Treatment of ALS

    Boros, B. D., Schoch, K. M., Kreple, C. J. & Miller, T., Jul 2022, In: Neurotherapeutics. 19, 4, p. 1145-1158 14 p.

    Research output: Contribution to journalReview articlepeer-review

    3 Scopus citations