Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.

by Richard S Finkel, Kevin M Flanigan, Brenda Wong, Carsten Bönnemann, Jacinda Sampson, H Lee Sweeney, Allen Reha, Valerie J Northcutt, Gary Elfring, Jay Barth, Stuart W Peltz
Published 2013-01-01

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

by Westley J Friesen, Briana Johnson, Jairo Sierra, Jin Zhuo, Priya Vazirani, Xiaojiao Xue, Yuki Tomizawa, Ramil Baiazitov, Christie Morrill, Hongyu Ren, Suresh Babu, Young-Choon Moon, Art Branstrom, Anna Mollin, Jean Hedrick, Josephine Sheedy, Gary Elfring, Marla Weetall, Joseph M Colacino, Ellen M Welch, Stuart W Peltz
Published 2018-01-01