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Development and psychometric analysis of the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT)

Journal Article published Dec 2015 in Neuromuscular Disorders volume 25 issue 12 on pages 937 to 944

Research funded by GlaxoSmithKline (HO-11-794) | European Commission (036825, 2012 3307) | Association Française contre les Myopathies (16104) | German Duchenne Parent Project | Deutsche Gesellschaft für Muskelkranke | Action Duchenne | Parent Project Muscular Dystrophy | Medical Research Council (98482)

Authors: Erik Landfeldt, Anna Mayhew, Michelle Eagle, Peter Lindgren, Christopher F. Bell, Michela Guglieri, Volker Straub, Hanns Lochmüller, Katharine Bushby

Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy

Journal Article published 10 Jul 2014 in Annals of Neurology volume 76 issue 4 on pages 541 to 549

Research funded by Action Duchenne | Charley's Fund | Cure Duchenne | Hope for Javier | Nash Avery Foundation | Zubin's Wish

Authors: Doris G. Leung, Daniel A. Herzka, W. Reid Thompson, Bing He, Genila Bibat, Gihan Tennekoon, Stuart D. Russell, Karl H. Schuleri, Albert C. Lardo, David A. Kass, Richard E. Thompson, Daniel P. Judge, Kathryn R. Wagner

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy

Journal Article published Feb 2019 in Life Science Alliance volume 2 issue 1 on page e201800186

Research funded by Foundation to Eradicate Duchenne | Clark Charitable Foundation | Action Duchenne | NIH (K99HL130035, R00HL130035, L40AR068727, T32AR056993) | U.S. Department of Defense (W81XWH-17-1-047) | CRI and the Intellectual and Developmental Disabilities Research Center Award (U54HD090257)

Authors: Christopher R Heier, Qing Yu, Alyson A Fiorillo, Christopher B Tully, Asya Tucker, Davi A Mazala, Kitipong Uaesoontrachoon, Sadish Srinivassane, Jesse M Damsker, Eric P Hoffman, Kanneboyina Nagaraju, Christopher F Spurney