Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children von Escolar, D. M., Henricson, E. K., Mayhew, J., Florence, J., Leshner, R., Patel, K. M., Clemens, P. R.

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Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids von McDonald, Craig M., Gordish-Dressman, Heather, Henricson, Erik K., Duong, Tina, Joyce, Nanette C., Jhawar, Sanjay, Leinonen, Mika, Hsu, Fengming, Connolly, Anne M., Cnaan, Avital, Abresch, Richard T., Dubrovsky, A., Kornberg, A., Ryan, M., Webster, R., Biggar, W.D., McAdam, L.C., Mah, J.K., Kolski, H., Vishwanathan, V., Chidambaranathan, S., Nevo, Y., Gorni, K., Carlo, J., Tulinius, M., Lotze, T., Bertorini, T.E., Day, J.W., Karachunski, P., Clemens, P.R., Abdel-Hamid, H., Teasley, J., Kuntz, N., Driscoll, S., Bodensteiner, J.B., Connolly, A.M., Pestronk, A., Abresch, R.T., Henricson, E.K., Joyce, N.C., McDonald, C.M., Cnaan, A., Morgenroth, L.P., Leshner, R., Tesi-Rocha, C., Thangarajh, M., Duong, T.

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Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim... von Mercuri, Eugenio, Osorio, Andrés Nascimento, Muntoni, Francesco, Buccella, Filippo, Desguerre, Isabelle, Kirschner, Janbernd, Tulinius, Már, de Resende, Maria Bernadete Dutra, Morgenroth, Lauren P., Gordish-Dressman, Heather, Johnson, Shelley, Kristensen, Allan, Werner, Christian, Trifillis, Panayiota, Henricson, Erik K., McDonald, Craig M.

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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study von Mercuri, Eugenio, Muntoni, Francesco, Osorio, Andrés Nascimento, Tulinius, Már, Buccella, Filippo, Morgenroth, Lauren P, Gordish-Dressman, Heather, Jiang, Joel, Trifillis, Panayiota, Zhu, Jin, Kristensen, Allan, Santos, Claudio L, Henricson, Erik K, McDonald, Craig M, Desguerre, Isabelle

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THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study von Mcdonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine M., Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart

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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicente... von McDonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart W.

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The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduce... von Henricson, Erik K., Abresch, R. Ted, Cnaan, Avital, Hu, Fengming, Duong, Tina, Arrieta, Adrienne, Han, Jay, Escolar, Diana M., Florence, Julaine M., Clemens, Paula R., Hoffman, Eric P., McDonald, Craig M.

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P.2.6 Comparison of 6MWD and person-reported functional measures in boys with Duchenne muscular dystrophy aged 4–12 years von Henricson, E.K, Abresch, R.T, Han, J.J, Nicorici, A, Goude, E.M, de Bie, E, McDonald, C.M

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P.2.9 The 6-min walk test and other endpoints in Duchenne MD: Multi center longitudinal natural history observations over 48 weeks von McDonald, C.M, Henricson, E.K, Abresch, R.T, Florence, J.M, Eagle, M, Gappmaier, E, Glanzman, A, Spiegel, R, Barth, J, Elfring, G, Reha, A, Peltz, S

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P.2.8 The 6-min walk test and clinical endpoints in Duchenne MD: Reliability, validity, and clinically-important differences von McDonald, C.M, Henricson, E.K, Abresch, R.T, Florence, J.M, Eagle, M, Gappmaier, E, Glanzman, A, Spiegel, R, Barth, J, Elfring, G, Reha, A, Peltz, S.W, P.T.C. Study Group

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P.2.4 Upper extremity reachable workspace evaluation in DMD using Kinect von Kurillo, G, Han, J.J, Nicorici, A, Johnson, L.B, Abresch, R.T, Henricson, E.K, McDonald, C.M, Bajcsy, R

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Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: the Cooperative International Neuromuscular Research Group (CINRG) von Escolar, Diana M, Henricson, Erik K, Pasquali, Livia, Gorni, Ksenija, Hoffman, Eric P

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A CINRG PILOT TRIAL OF OXATOMIDE IN STEROID-NAÏVE DUCHENNE MUSCULAR DYSTROPHY von Buyse, GM, Escolar, DM, Goemans, N, Henricson, EK, van den Hauwe, M, Vallejos, AJ, Shao, C, Patel, KM, McCarter, R, Leshner, R, Florence, J, Mayhew, J

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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study von Mercuri, E, Muntoni, F, Osorio, AN, Tulinius, M, Buccella, F, Morgenroth, LP, Gordish-Dressman, H, Jiang, J, Trifillis, P, Zhu, J, Kristensen, A, Santos, CL, Henricson, EK, McDonald, CM, Desguerre, I, STRIDE, CINRG Duchenne Natural History Investigators

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Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study von McDonald, Craig M, Henricson, Erik K, Abresch, Richard T, Duong, Tina, Joyce, Nanette C, Hu, Fengming, Clemens, Paula R, Hoffman, Eric P, Cnaan, Avital, Gordish-Dressman, Heather, Vishwanathan, Vijay, Chidambaranathan, S, Biggar, W. Douglas, McAdam, Laura C., Mah, Jean K., Tulinius, Mar, Cnaan, Avital, Morgenroth, Lauren P., Leshner, Robert, Tesi-Rocha, Carolina, Thangarajh, Mathula, Duong, Tina, Kornberg, Andrew, Ryan, Monique, Nevo, Yoram, Dubrovsky, Alberto, Clemens, Paula R., Abdel-Hamid, Hoda, Connolly, Anne M., Pestronk, Alan, Teasley, Jean, Bertorin, Tulio E., Webster, Richard, Kolski, Hanna, Kuntz, Nancy, Driscoll, Sherilyn, Bodensteiner, John B., Carlo, Jose, Gorni, Ksenija, Lotze, Timothy, Day, John W., Karachunski, Peter, Henricson, Erik K., Abresch, Richard T., Joyce, Nanette C., McDonald, Craig M.

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Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial von 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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Better implant survival with modern ankle prosthetic designs: 1,226 total ankle prostheses followed for up to 20 years in the Swedish Ankle Registry von Undén, Alexandra, Jehpsson, Lars, Kamrad, Ilka, Carlsson, Åke, Henricson, Anders, Karlsson, Magnus K, Rosengren, Björn E

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DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study von Bello, Luca, Morgenroth, Lauren P, Gordish-Dressman, Heather, Hoffman, Eric P, McDonald, Craig M, Cirak, Sebahattin

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X-linked Malformation Deafness: Neurodevelopmental Symptoms Are Common in Children With IP3 Malformation and Mutation in POU3F4 von Smeds, Henrik, Wales, Jeremy, Karltorp, Eva, Anderlid, Britt-Marie, Henricson, Cecilia, Asp, Filip, Anmyr, Lena, Lagerstedt-Robinson, Kristina, Löfkvist, Ulrika

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Development of a patient‐reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM von Klingels, K, Mayhew, A G, Mazzone, E S, Duong, T, Decostre, V, Werlauff, U, Vroom, E, Mercuri, E, Goemans, N M, Eagle, M, De Groot, I, Main, M, Messina, S, Campion, G, Servais, L, Van den Hauwe, M, Es, E, Pane, M, Buccella, F, Kuijer, J, Ceradini, F, Henricson, E, McDonald, C, Florence, J

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