PhysiologyPLoS CurrentsPeer-reviewed
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…·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.
DOI 10.1371/currents.md.e1e8f2be7c949f9ffe81ec6fca1cce6a