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

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Spirometry correlates with physical activity in patients with Duchenne muscular dystrophy by Kaslow, Jacob A., Sokolow, Andrew G., Donnelly, Thomas, Buchowski, Maciej S., Markham, Larry W., Burnette, William Bryan, Soslow, Jonathan. H.

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Developing standardized corticosteroid treatment for Duchenne muscular dystrophy by Guglieri, Michela, Bushby, Kate, McDermott, Michael P, Hart, Kimberly A, Tawil, Rabi, Martens, William B, Herr, Barbara E, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michele, Brown, Mary W, Willis, Tracey, Hirtz, Deborah, Shieh, Perry B, Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Butterfield, Russell J, Horrocks, Iain, Spinty, Stefan, Flanigan, Kevin M, Kuntz, Nancy L, Baranello, Giovanni, Roper, Helen, Morrison, Leslie, Mah, Jean K, Manzur, Adnan Y, McDonald, Craig M, Schara, Ulrike, von der Hagen, Maja, Barohn, Richard J, Campbell, Craig, Darras, Basil T, Finkel, Richard S, Vita, Giuseppe, Hughes, Imelda, Mongini, Tiziana, Pegoraro, Elena, Wicklund, Matthew, Wilichowski, Ekkehard, Bryan Burnette, W, Howard, James F, McMillan, Hugh J, Thangarajh, Mathula, Griggs, Robert C

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Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial by Guglieri, Michela, Bushby, Kate, McDermott, Michael P, Hart, Kimberly A, Tawil, Rabi, Martens, William B, Herr, Barbara E, McColl, Elaine, Speed, Chris, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michelle, Brown, Mary W, Willis, Tracey, Griggs, Robert C, Straub, Volker, van Ruiten, Henriette, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B, Spinty, Stefan, Maggi, Lorenzo, Baranello, Giovanni, Butterfield, Russell J, Horrocks, I. A, Roper, Helen, Alhaswani, Zoya, Flanigan, Kevin M, Kuntz, Nancy L, Manzur, Adnan, Darras, Basil T, Kang, Peter B, Morrison, Leslie, Krzesniak-Swinarska, Monika, Mah, Jean K, Mongini, Tiziana E, Ricci, Federica, von der Hagen, Maja, Finkel, Richard S, O’Reardon, Kathleen, Wicklund, Matthew, Kumar, Ashutosh, McDonald, Craig M, Han, Jay J, Joyce, Nanette, Henricson, Erik K, Schara-Schmidt, Ulrike, Gangfuss, Andrea, Wilichowski, Ekkehard, Barohn, Richard J, Statland, Jeffrey M, Campbell, Craig, Vita, Giuseppe, Vita, Gian Luca, Howard, James F, Hughes, Imelda, McMillan, Hugh J, Pegoraro, Elena, Bello, Luca, Burnette, W. Bryan, Thangarajh, Mathula, Chang, Taeun

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A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial by Crow, Rebecca A, Hart, Kimberly A, McDermott, Michael P, Tawil, Rabi, Martens, William B, Herr, Barbara E, McColl, Elaine, Wilkinson, Jennifer, Kirschner, Janbernd, King, Wendy M, Eagle, Michele, Brown, Mary W, Hirtz, Deborah, Lochmuller, Hanns, Straub, Volker, Ciafaloni, Emma, Shieh, Perry B, Spinty, Stefan, Childs, Anne-Marie, Manzur, Adnan Y, Morandi, Lucia, Butterfield, Russell J, Horrocks, Iain, Roper, Helen, Flanigan, Kevin M, Kuntz, Nancy L, Mah, Jean K, Morrison, Leslie, Darras, Basil T, von der Hagen, Maja, Schara, Ulrike, Wilichowski, Ekkehard, Mongini, Tiziana, McDonald, Craig M, Vita, Giuseppe, Barohn, Richard J, Finkel, Richard S, Wicklund, Matthew, McMillan, Jr, Hugh J, Hughes, Imelda, Pegoraro, Elena, Bryan Burnette, W, Howard, James F, Thangarajh, Mathula, Campbell, Craig, Griggs, Robert C, Bushby, Kate, Guglieri, Michela

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Disease Control and Safety of Belimumab Plus Standard Therapy Over 7 Years in Patients with Systemic Lupus Erythematosus by GINZLER, Ellen M, WALLACE, Daniel J, FREIMUTH, William W, PETRI, Michelle A, MERRILL, Joan T, FURIE, Richard A, STOHL, William, CHATHAM, W. Winn, WEINSTEIN, Arthur, MCKAY, James D, MCCUNE, W. Joseph, ZHONG, Z. John

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Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy by Campbell, Craig, McColl, Elaine, McDermott, Michael P., Martens, William B., Guglieri, Michela, Griggs, Robert C., Straub, Volker, Childs, Anne-Marie, Ciafaloni, Emma, Shieh, Perry B., Spinty, Stefan, Butterfield, Russell J., Horrocks, Iain, Roper, Helen, Maggi, Lorenzo, Baranello, Giovanni, Flanigan, Kevin M., Kuntz, Nancy L., Manzur, Adnan Y., Darras, Basil T., Kang, Peter, Mah, Jean K., Mongini, Tiziana, Ricci, Federica, Morrison, Leslie, Krzesniak-Swinarska, Monika, von der Hagen, Maja, Finkel, Richard S., Kumar, Ashutosh, Wicklund, Matthew, McDonald, Craig M., Henricson, Erik K., Schara-Schmidt, Ulrike, Wilichowski, Ekkehard, Barohn, Richard J., Statland, Jeffrey, Kirschner, Janbernd, Vita, Giuseppe, Vita, Gian Luca, Howard, James F., Hughes, Imelda, McMillan, Hugh J., Pegoraro, Elena, Bello, Luca, Burnette, W. Bryan, Thangarajh, Mathula, Chang, Taeun

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