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Personal profile

Research interests

My research program focuses on:

1. Gene down regulation strategies (RNAi and antisense oligonucleotides) to understand disease pathogenesis and provide novel therapies for neurodegenerative diseases, in particular amyotrophic lateral sclerosis (ALS)

2. How dysfunction of mitochondria causes loss of motor neurons.

Areas of Clinical Interest

ALS and neurological disorders

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  • 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
    2 Scopus citations
  • Is presymptomatic ALS perivascular?

    Kreple, C., Schoch, K. & Miller, T. M., Apr 2021, In: Nature medicine. 27, 4, p. 585-586 2 p.

    Research output: Contribution to journalComment/debate

  • Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis

    Paganoni, S., Hendrix, S., Dickson, S. P., Knowlton, N., Macklin, E. A., Berry, J. D., Elliott, M. A., Maiser, S., Karam, C., Caress, J. B., Owegi, M. A., Quick, A., Wymer, J., Goutman, S. A., Heitzman, D., Heiman-Patterson, T. D., Jackson, C. E., Quinn, C., Rothstein, J. D., Kasarskis, E. J. & 41 others, Katz, J., Jenkins, L., Ladha, S., Miller, T. M., Scelsa, S. N., Vu, T. H., Fournier, C. N., Glass, J. D., Johnson, K. M., Swenson, A., Goyal, N. A., Pattee, G. L., Andres, P. L., Babu, S., Chase, M., Dagostino, D., Hall, M., Kittle, G., Eydinov, M., McGovern, M., Ostrow, J., Pothier, L., Randall, R., Shefner, J. M., Sherman, A. V., St Pierre, M. E., Tustison, E., Vigneswaran, P., Walker, J., Yu, H., Chan, J., Wittes, J., Yu, Z. F., Cohen, J., Klee, J., Leslie, K., Tanzi, R. E., Gilbert, W., Yeramian, P. D., Schoenfeld, D. & Cudkowicz, M. E., Jan 2021, In: Muscle and Nerve. 63, 1, p. 31-39 9 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access
    5 Scopus citations
  • Pathogenic Huntingtin Repeat Expansions in Patients with Frontotemporal Dementia and Amyotrophic Lateral Sclerosis

    The American Genome Center (TAGC), The FALS Sequencing Consortium, The Genomics England Research Consortium, The International ALS/FTD Genomics Consortium (iAFGC), The International FTD Genetics Consortium (IFGC), The International LBD Genomics Consortium (iLBDGC), The NYGC ALS Consortium, the University of Maryland Brain and Tissue Bank and NIH NeuroBioBank & The PROSPECT Consortium, Feb 3 2021, In: Neuron. 109, 3, p. 448-460.e4

    Research output: Contribution to journalArticlepeer-review

    3 Scopus citations
  • Smartphone data during the COVID-19 pandemic can quantify behavioral changes in people with ALS

    Beukenhorst, A. L., Collins, E., Burke, K. M., Rahman, S. M., Clapp, M., Konanki, S. C., Paganoni, S., Miller, T. M., Chan, J., Onnela, J. P. & Berry, J. D., Feb 2021, In: Muscle and Nerve. 63, 2, p. 258-262 5 p.

    Research output: Contribution to journalArticlepeer-review

    Open Access