Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy

بواسطة Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Mollin, Anna, Sheedy, Josephine, Yeh, Shirley, Petruska, Janet, Narasimhan, Jana, Dakka, Amal, Welch, Ellen M., Karp, Gary, Chen, Karen S., Metzger, Friedrich, Ratni, Hasane, Lotti, Francesco, Tisdale, Sarah, Naryshkin, Nikolai A., Pellizzoni, Livio, Paushkin, Sergey, Ko, Chien-Ping, Weetall, Marla
الحاوية / القاعدة Hum Mol Genet (2016)

Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties

بواسطة Cao*, Liangxian, Weetall, Marla, Trotta, Christopher, Cintron, Katherine, Ma, Jiyuan, Kim, Min Jung, Furia, Bansri, Romfo, Charles, Graci, Jason D., Li, Wencheng, Du, Joshua, Sheedy, Josephine, Hedrick, Jean, Risher, Nicole, Yeh, Shirley, Qi, Hongyan, Arasu, Tamil, Hwang, Seongwoo, Lennox, William, Kong, Ronald, Petruska, Janet, Moon, Young-Choon, Babiak, John, Davis, Thomas W., Jacobson, Allan, Almstead, Neil G., Branstrom, Art, Colacino, Joseph M., Peltz, Stuart W.
الحاوية / القاعدة Mol Cancer Ther (2018)