325P Is upper extremity contracture progression related to changes in upper extremity function in Duchenne muscular dystrophy? An international multicenter natural history study von Van Der Holst, M., Italianer, M., Wolfe, A., Chesshyre, M., Voit, T., Straub, V., de Lucia, S., Servais, L., Hogrel, J., Pelsma, M., de Groot, I., Houwen, S., Muntoni, F., Niks, E.

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DMD – BIOMARKERS & OUTCOME MEASURES von Main, M., Waters, K., Abbott, L., Longatto, J., Chesshyre, M., Baranello, G., Muntoni, F.

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DMD – BIOMARKERS & OUTCOME MEASURES: P.128 Developing a behavioural assessment tool to monitor compliance with physical assessment in Duchenne muscular dystrophy von Main, M., Waters, K., Abbott, L., Longatto, J., Chesshyre, M., Baranello, G., Muntoni, F.

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P.126 Genotype-unmatched controls are feasible for drug development in Duchenne muscular dystrophy (DMD) von Muntoni, F., Signorovitch, J., Frean, M., Fillbrunn, M., Sajeev, G., Ward, S., McDonald, C., Goemans, N., Niks, E., Wong, B., Servais, L., Straub, V., de Groot, I., Chesshyre, M., Tian, C., Manzur, A., Mercuri, E., Aartsma-Rus, A., Groups, Study

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DMD & BMD – CLINICAL von Gupta, V. Ayyar, Abbott, L., Chesshyre, M., Main, M., Baranello, G., Scoto, M., Manzur, A., Muntoni, F., Clinical Network, UK Northstar

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DMD & BMD – CLINICAL: P.59 Functional progression in young DMD von Gupta, V. Ayyar, Abbott, L., Chesshyre, M., Main, M., Baranello, G., Scoto, M., Manzur, A., Muntoni, F., Clinical Network, UK Northstar

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DMD & BMD – CLINICAL: P.58 Hypermobility in young boys with Duchenne muscular dystrophy and its effect on attainment of North Star Ambulatory Assessment functional skills von Abbott, L., Selby, V., Gupta, V. Ayyar, Wadsworth, S., Wolfe, A., Chesshyre, M., Baranello, G., Scoto, M., Manzur, A., Main, M., Muntoni, F.

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DMD & BMD – CLINICAL von Abbott, L., Selby, V., Gupta, V. Ayyar, Wadsworth, S., Wolfe, A., Chesshyre, M., Baranello, G., Scoto, M., Manzur, A., Main, M., Muntoni, F.

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Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation von Stimpson, G, Chesshyre, M, Baranello, G, Muntoni, F

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Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy von Gupta, V.A., Pitchforth, J.M., Domingos, J., Ridout, D., Iodice, M., Rye, C., Chesshyre, M., Wolfe, A., Selby, V., Mayhew, A., Mazzone, E.S., Ricotti, V., Hogrel, J.Y., Niks, E.H., Groot, I. de, Servais, L., Straub, V., Mercuri, E., Manzur, A.Y., Muntoni, F.

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DMD genotypes and motor function in Duchenne muscular dystrophy von Muntoni, F., Signorovitch, J., Sajeev, G., Lane, H., Jenkins, M., Dieye, I., Ward, S.J., McDonald, C., Goemans, N., Niks, E.H., Wong, B.D., Servais, L., Straub, V., Guglieri, M., Groot, I.J.M. de, Chesshyre, M., Tian, C.X., Manzur, A.Y., Mercuri, E., Aartsma-Rus, A.

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Genotype-related respiratory progression in Duchenne muscular dystrophy: a multicenter international study von Trucco, F., Ridout, D., Domingos, J., Maresh, K., Chesshyre, M., Munot, P., Sarkozy, A., Robb, S., Quinlivan, R., Riley, M., Wallis, C., Chan, E., Abel, F., Lucia, S. de, Hogrel, J.Y., Niks, E.H., Groot, I. de, Servais, L., Straub, V., Ricotti, V., Manzur, A., Muntoni, F.

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North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up von Coratti, G., Pane, M., Brogna, C., Ricotti, V., Messina, S., D'Amico, A., Bruno, C., Vita, G., Berardinelli, A., Mazzone, E., Magri, F., Ricci, F., Mongini, T., Battini, R., Bello, L., Pegoraro, E., Baranello, G., Previtali, S.C., Politano, L., Comi, G.P., Sansone, V.A., Donati, A., Hogrel, J.Y., Straub, V., Lucia, S. de, Niks, E., Servais, L., Groot, I. de, Chesshyre, M., Bertini, E., Goemans, N., Muntoni, F., Mercuri, E.

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North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up von Coratti, G, Pane, M, Brogna, C, Ricotti, V, Messina, S, D'Amico, A, Bruno, C, Vita, G, Berardinelli, A, Mazzone, E, Magri, F, Ricci, F, Mongini, T, Battini, R, Bello, L, Pegoraro, E, Baranello, G, Previtali, SC, Politano, L, Comi, GP, Sansone, VA, Donati, A, Hogrel, JY, Straub, V, De Lucia, S, Niks, E, Servais, L, De Groot, I, Chesshyre, M, Bertini, E, Goemans, N, Muntoni, F, Mercuri, E, on behalf on the International DMD Group and the iMDEX Consortiu

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Dysphagia due to left atrial enlargment after mitral Starr valve replacement von Chesshyre, M H, Braimbridge, M V

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Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy von Ayyar Gupta, Vandana, Pitchforth, Jacqueline M, Domingos, Joana, Ridout, Deborah, Iodice, Mario, Rye, Catherine, Chesshyre, Mary, Wolfe, Amy, Selby, Victoria, Mayhew, Anna, Mazzone, Elena S, Ricotti, Valeria, Hogrel, Jean-Yves, Niks, Erik H, de Groot, Imelda, Servais, Laurent, Straub, Volker, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco

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DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials von Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Lane, Henry, Jenkins, Madeline, Dieye, Ibrahima, Ward, Susan J., McDonald, Craig, Goemans, Nathalie, Niks, Erik H., Wong, Brenda, Servais, Laurent, Straub, Volker, Guglieri, Michela, de Groot, Imelda J.M., Chesshyre, Mary, Tian, Cuixia, Manzur, Adnan Y, Mercuri, Eugenio, Aartsma-Rus, Annemieke

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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping von Mercuri, E., Seferian, A.M., Servais, L., Deconinck, N., Stevenson, H., Ni, X., Zhang, W., East, L., Yonren, S., Muntoni, F., Deconinck, Nicolas, Van Coster, Rudy, Vanlander, Arnaud, Seferian, Andreea, De Lucia, Silvana, Gidaro, Teresa, Brande, Laura Vanden, Servais, Laurent, Kirschner, Janbernd, Borell, Sabine, Mercuri, Eugenio, Brogna, Claudia, Pane, Marika, Fanelli, Lavinia, Norcia, Giulia, Muntoni, Francesco, Brusa, Chiara, Chesshyre, Mary, Maresh, Kate, Pitchforth, Jaqueline, Schottlaender, Lucia, Scoto, Mariacristina, Silwal, Arpana, Trucco, Fedrica

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Determining minimal clinically important differences in the North Star Ambulatory Assessment von Ayyar Gupta, Vandana, Pitchforth, Jacqueline M, Domingos, Joana, Ridout, Deborah, Iodice, Mario, Rye, Catherine, Chesshyre, Mary, Wolfe, Amy, Selby, Victoria, Mayhew, Anna, Mazzone, Elena S, Ricotti, Valeria, Hogrel, Jean-Yves, Niks, Erik H, de Groot, Imelda, Servais, Laurent, Straub, Volker, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco

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Improving empiric antibiotic prescribing in pediatric bloodstream infections: a potential application of weighted-incidence syndromic combination antibiograms (WISCA) von Cook, A, Sharland, M, Yau, Y, Bielicki, J

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