Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits
Richard T Wang, Cheri A Silverstein Fadlon, J Wes Ulm, Ivana Jankovic, Ascia Eskin, Ake Lu, Vanessa Miller, Rita M Cantor, Ning Li, Robert Elashoff, Ann S Martin, Holly Peay, Stanley F Nelson
This study uses online self-report data from DuchenneConnect to show that corticosteroid use delays loss of ambulation in DMD, with deflazacort more effective than prednisone.
bioRxiv · Dec 8, 2014Physiology