Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia

por Branstrom, Arthur, Cao, Liangxian, Furia, Bansri, Trotta, Christopher, Santaguida, Marianne, Graci, Jason D., Colacino, Joseph M., Ray, Balmiki, Li, Wencheng, Sheedy, Josephine, Mollin, Anna, Yeh, Shirley, Kong, Ronald, Sheridan, Richard, Baird, John D., O’Keefe, Kylie, Spiegel, Robert, Goodwin, Elizabeth, Keating, Suzanne, Weetall, Marla
Publicado 2022

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

por 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
Publicado 2016

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

por Friesen, Westley J., Johnson, Briana, Sierra, Jairo, Zhuo, Jin, Vazirani, Priya, Xue, Xiaojiao, Tomizawa, Yuki, Baiazitov, Ramil, Morrill, Christie, Ren, Hongyu, Babu, Suresh, Moon, Young-Choon, Branstrom, Art, Mollin, Anna, Hedrick, Jean, Sheedy, Josephine, Elfring, Gary, Weetall, Marla, Colacino, Joseph M., Welch, Ellen M., Peltz, Stuart W.
Publicado 2018

Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia

por Morini, Elisabetta, Chekuri, Anil, Logan, Emily M., Bolduc, Jessica M., Kirchner, Emily G., Salani, Monica, Krauson, Aram J., Narasimhan, Jana, Gabbeta, Vijayalakshmi, Grover, Shivani, Dakka, Amal, Mollin, Anna, Jung, Stephen P., Zhao, Xin, Zhang, Nanjing, Zhang, Sophie, Arnold, Michael, Woll, Matthew G., Naryshkin, Nikolai A., Weetall, Marla, Slaugenhaupt, Susan A.
Publicado 2023

Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent

por Jernigan, Finith, Branstrom, Arthur, Baird, John D., Cao, Liangxian, Dali, Mandar, Furia, Bansri, Kim, Min Jung, O'Keefe, Kylie, Kong, Ronald, Laskin, Oscar L., Colacino, Joseph M., Pykett, Mark, Mollin, Anna, Sheedy, Josephine, Dumble, Melissa, Moon, Young-Choon, Sheridan, Richard, Mühlethaler, Tobias, Spiegel, Robert J., Prota, Andrea E., Steinmetz, Michel O., Weetall, Marla
Publicado 2021