Grace Liu, Philip Lipari, Anna Mollin, Stephen Jung, Irina Teplova, Wencheng Li, Lanqing Ying, Vijay More, William Lennox, Shirley Yeh, Eric McGann, Young-Choon Moon, Cari Rice, Eduardo Huarte, Barbara Gruszka, Balmiki Ray, Elizabeth Goodwin, Patricia Buckendahl, Edward Yurkow, Bruce Braughton, Jana Narasimhan, Ellen Welch, Gregory Voronin, Marla Weetall
Duchenne muscular dystrophy (DMD) is a progressive disabling X-linked recessive disorder that causes gradual and irreversible loss of muscle, resulting in early death. The corticosteroids prednisone/prednisolone and deflazacort are used to treat DMD as the standard of care; however, only deflazacort is FDA approved for DMD. The novel atypical corticosteroid vamorolone is being investigated for treatment of DMD. We compared the pharmaceutical properties as well as the efficacy and safety of the three corticosteroids across multiple doses in the B10-mdx DMD mouse model...
October 11, 2023: Human Molecular Genetics