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

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

Protein production is an early biomarker for RNA‐targeted therapies

door 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.
Gepubliceerd in Ann Clin Transl Neurol (2018)

In vivo kinetic approach reveals slow SOD1 turnover in the CNS

door 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.
Gepubliceerd in J Clin Invest (2015)

Defining SOD1 ALS natural history to guide therapeutic clinical trial design

door 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
Gepubliceerd in J Neurol Neurosurg Psychiatry (2016)