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.
Clinical interests
ALS and neurological disorders
Available to Mentor:
- PhD/MSTP Students
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MiToS and King’s staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trial
Gebrehiwet, P., Meng, L., Rudnicki, S. A., Sarocco, P., Wei, J., Wolff, A. A., Chiò, A., Andrews, J. A., Genge, A., Jackson, C. E., Lechtzin, N., Miller, T. M. & Shefner, J. M., 2023, In: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 24, 3-4, p. 304-310 7 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Protective Effects of Lovastatin in a Population-Based ALS Study and Mouse Model
Kreple, C. J., Searles Nielsen, S., Schoch, K. M., Shen, T., Shabsovich, M., Song, Y., Racette, B. A. & Miller, T. M., May 2023, In: Annals of neurology. 93, 5, p. 881-892 12 p.Research output: Contribution to journal › Article › peer-review
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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 1 2022, In: Annals of neurology. 91, 2, p. 165-175 11 p.Research output: Contribution to journal › Article › peer-review
Open Access20 Scopus citations -
Amyotrophic Lateral Sclerosis Clinical Trials and Interpretation of Functional End Points and Fluid Biomarkers: A Review
Shefner, J. M., Bedlack, R., Andrews, J. A., Berry, J. D., Bowser, R., Brown, R., Glass, J. D., Maragakis, N. J., Miller, T. M., Rothstein, J. D. & Cudkowicz, M. E., Dec 12 2022, In: JAMA Neurology. 79, 12, p. 1312-1318 7 p.Research output: Contribution to journal › Review article › peer-review
2 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 others, Feb 2022, In: Nature neuroscience. 25, 2, p. 226-237 12 p.Research output: Contribution to journal › Article › peer-review
Open Access26 Scopus citations