2342 Protein production as an early pharmacodynamics biomarker for RNA-targeting therapies

per Self, Wade K., Schoch, Kathleen, Bollinger, James, Cole, Tracy, Kordasiewicz, Holly, Bateman, Randall, Miller, Timothy
Publicat a J Clin Transl Sci (2018)

Protein production is an early biomarker for RNA‐targeted therapies

per Self, Wade K., Schoch, Kathleen M., Alex, Jacob, Barthélemy, Nicolas, Bollinger, James G., Sato, Chihiro, Cole, Tracy, Kordasiewicz, Holly B., Swayze, Eric, Bateman, Randall J., Miller, Timothy M.
Publicat a Ann Clin Transl Neurol (2018)

In vivo kinetic approach reveals slow SOD1 turnover in the CNS

per Crisp, Matthew J., Mawuenyega, Kwasi G., Patterson, Bruce W., Reddy, Naveen C., Chott, Robert, Self, Wade K., Weihl, Conrad C., Jockel-Balsarotti, Jennifer, Varadhachary, Arun S., Bucelli, Robert C., Yarasheski, Kevin E., Bateman, Randall J., Miller, Timothy M.
Publicat a J Clin Invest (2015)

Defining SOD1 ALS natural history to guide therapeutic clinical trial design

per Bali, Taha, Self, Wade, Liu, Jingxia, Siddique, Teepu, Wang, Leo H, Bird, Thomas D, Ratti, Elena, Atassi, Nazem, Boylan, Kevin B, Glass, Jonathan D, Maragakis, Nicholas J, Caress, James B, McCluskey, Leo F, Appel, Stanley H, Wymer, James P, Gibson, Summer, Zinman, Lorne, Mozaffar, Tahseen, Callaghan, Brian, McVey, April L, Jockel-Balsarotti, Jennifer, Allred, Peggy, Fisher, Elena R, Lopate, Glenn, Pestronk, Alan, Cudkowicz, Merit E, Miller, Timothy M
Publicat a J Neurol Neurosurg Psychiatry (2016)