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Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L by Ivana F. Audhya, Shelagh M. Szabo, Andrea Bever, Fiona O’Sullivan, Daniel C. Malone, David Feeny, Peter Neumann, Susan T. Iannaccone, P. Jayasinghe, Katherine L. Gooch

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Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy by Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac

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Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1 by Preeti Kumari, Lauren M. Sullivan, Zhaozhi Li, E. Parker Conquest, Elizabeth Cornforth, Rojashree Jayakumar, Ningyan Hu, J. Alexander Sizemore, Brigham B. McKee, Robert R. Kitchen, Paloma González-Pérez, Constance Linville, Karla Castro, Hilda Gutierrez, Soleil Samaan, Elise L. Townsend, Basil T. Darras, Seward B. Rutkove, Susan T. Iannaccone, Paula R. Clemens, Araya Puwanant, Sudeshna Das, Thurman M. Wheeler

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BET1 variants establish impaired vesicular transport as a cause for muscular dystrophy with epilepsy by Sandra Donkervoort, Niklas Krause, Mykola Dergai, Pomi Yun, Judith Koliwer, Svetlana Gorokhova, Janelle Geist Hauserman, Beryl B Cummings, Ying Hu, Rosemarie Smith, Prech Uapinyoying, Vijay S Ganesh, Partha S Ghosh, Kristin G Monaghan, Seby L Edassery, Pia E Ferle, Sarah Silverstein, Katherine R Chao, Molly Snyder, Sara Ellingwood, Diana Bharucha‐Goebel, Susan T Iannaccone, Matteo Dal Peraro, A Reghan Foley, Jeffrey N Savas, Véronique Bolduc, Dirk Fasshauer, Carsten G Bönnemann, Michael Schwake

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Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians by Aravindhan Veerapandiyan, Anne M. Connolly, Katherine D. Mathews, Stanley Nelson, Craig McDonald, Richard S. Finkel, Vettaikorumakankav Vedanarayanan, Cuixia Tian, Susan Apkon, Julie A. Parsons, Jonathan H. Soslow, William Bryan Burnette, Kaitlin Y. Batley, Susan T. Iannaccone, Carolina Tesi Rocha, Kevin M. Flanigan, Diana Bharucha‐Goebel, Sarah Wright, Migvis Monduy, Simona Treidler, Ashutosh Kumar, Nancy L. Kuntz, Vamshi K. Rao, Rachel Schrader, Saunder M. Bernes, Vikki Ann Stefans, Jena M. Krueger, Marcia V. Felker, Omer Abdul Hamid, Arpita Lakhotia, Susan Matesanz, Partha S. Ghosh, Natalie Katz, Hoda Abdel‐Hamid, Chamindra G. Laverty, Bo Hoon Lee, Amy Harper, Leigh Ramos‐Platt, Diana Castro, Russell J. Butterfield, Crystal M. Proud, Craig M. Zaidman, Emma Ciafaloni

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