Research Output per year
Research Output per year
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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Research Output
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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, , Jan 2021, In: Muscle and Nerve. 63, 1, p. 31-39 9 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Amyotrophic lateral sclerosis care and research in the United States during the COVID-19 pandemic: Challenges and opportunities
Andrews, J. A., Berry, J. D., Baloh, R. H., Carberry, N., Cudkowicz, M. E., Dedi, B., Glass, J., Maragakis, N. J., Miller, T. M., Paganoni, S., Rothstein, J. D., Shefner, J. M., Simmons, Z., Weiss, M. D. & Bedlack, R. S., Jan 1 2020, (Accepted/In press) In: Muscle and Nerve.Research output: Contribution to journal › Article › peer-review
Open Access4 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, , 2020, (Accepted/In press) In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.Research output: Contribution to journal › Article › peer-review
Open Access -
A viral toolkit for recording transcription factor-DNA interactions in live mouse tissues
Cammack, A. J., Moudgil, A., Chen, J., Vasek, M. J., Shabsovich, M., McCullough, K., Yen, A., Lagunas, T., Maloney, S. E., He, J., Chen, X., Hooda, M., Wilkinson, M. N., Miller, T. M., Mitra, R. D. & Dougherty, J. D., May 5 2020, In: Proceedings of the National Academy of Sciences of the United States of America. 117, 18, p. 10003-10014 12 p.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
Burst mitofusin activation reverses neuromuscular dysfunction in murine cmt2a
Franco, A., Dang, X., Walton, E. K., Ho, J. N., Zablocka, B., Ly, C., Miller, T. M., Baloh, R. H., Shy, M. E., Yoo, A. S. & Dorn, G. W., Oct 2020, In: eLife. 9, p. 1-42 42 p., e61119.Research output: Contribution to journal › Article › peer-review
Open Access