Reliability of the North Star Ambulatory Assessment in a multicentric setting von Mazzone, E.S, Messina, S, Vasco, G, Main, M, Eagle, M, D’Amico, A, Doglio, L, Politano, L, Cavallaro, F, Frosini, S, Bello, L, Magri, F, Corlatti, A, Zucchini, E, Brancalion, B, Rossi, F, Ferretti, M, Motta, M.G, Cecio, M.R, Berardinelli, A, Alfieri, P, Mongini, T, Pini, A, Astrea, G, Battini, R, Comi, G, Pegoraro, E, Morandi, L, Pane, M, Angelini, C, Bruno, C, Villanova, M, Vita, G, Donati, M.A, Bertini, E, Mercuri, E

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Interim analysis of the phase 3 CHERISH study evaluating nusinersen in patients with later-onset spinal muscular atrophy (SMA): Primary and descriptive secondary endpoints von Mercuri, E, Finkel, R, Kirschner, J, Chiriboga, C.A, Kuntz, N, Darras, B, Shieh, P.B, Saito, K, De Vivo, D.C, Mazzone, E.S, Montes, J, Yang, Q, Zhong, Z.J, Gheuens, S, Bennett, C.F, Schneider, E, Farwell, W

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T.P.37 von Mazzone, E.S, Mayhew, A, Main, M, Montes, J, Ramsey, D, Scoto, M, Pane, M, Bushby, K, Finkel, R, Muntoni, F, Mercuri, E

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T.P.11 von Pane, M, Mazzone, E.S, Fanelli, L, Sanctis, R. De, Palermo, C, Sivo, S, D’Amico, A, Messina, S, Politano, L, Battini, E, Pedemonte, M, Pegoraro, E, Berardinelli, A.L, D’Angelo, G, Pini, A, Baranello, G, Mercuri, E

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T.P.4 von Mazzone, E.S, Pane, M, Sivo, S, Palermo, C, Sormani, M.P, Messina, S, D’Amico, A, Vita, G.L, Fanelli, L, Berardinelli, A, Donati, M.A, Baranello, G, Battini, R, Pegoraro, E, Politano, L, Bruno, C, Comi, G.P, Bertini, E, Mercuri, E

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S.P.4 Functional changes in Duchenne muscular dystrophy: A 24 month longitudinal cohort study von Mazzone, E.S, Pane, M, Scalise, R, Sormani, M.P, Torrente, Y, Berardinelli, A, Messina, S, Cavallaro, F, Amico, A. D, Doglio, L, Bruno, C, Politano, L, Frosini, S, Battini, R, Bello, L, Pegoraro, E, Bonfiglio, S, Comi, G, Pini, A, de Sanctis, R, Bianco, F, Vasco, G, Mongini, T, Donati, M.A, Previtali, S, Napolitano, S, Vita, G, Bertini, E, Mercuri, E

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M.P.2.01 Reliability of the north star ambulatory assessment in a multicentric setting von Mazzone, E.S, Messina, S, Vasco, G, Main, M, Eagle, M, D’Amico, A, Bruno, C, Politano, L, Berardinelli, A, Mongini, T, Pini, A, Battini, R, Comi, G, Pegoraro, E, Morandi, L, Villanova, M, Vita, G, Bertini, E, Mercuri, E

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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, Katrijn, Mayhew, A.G, Mazzone, E.S, Duong, T, Decostre, V, Werlauff, U, Vroom, E, Mercuri, E, Goemans, Nathalie, The Upper Limb Clinical Outcome Group

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T.P.37: Old measures and new analysis in non ambulant SMA patients von Mazzone, E.S., Mayhew, A., Main, M., Montes, J., Ramsey, D., Scoto, M., Pane, M., Bushby, K., Finkel, R., Muntoni, F., Mercuri, E.

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T.P.4: Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36 month changes von Mazzone, E.S., Pane, M., Sivo, S., Palermo, C., Sormani, M.P., Messina, S., D’Amico, A., Vita, G.L., Fanelli, L., Berardinelli, A., Donati, M.A., Baranello, G., Battini, R., Pegoraro, E., Politano, L., Bruno, C., Comi, G.P., Bertini, E., Mercuri, E.

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T.P.11: Longitudinal assessment of Upper Limb function in DMD patients: 12 month changes von Pane, M., Mazzone, E.S., Fanelli, L., Sanctis, R. De, Palermo, C., Sivo, S., D’Amico, A., Messina, S., Politano, L., Battini, E., Pedemonte, M., Pegoraro, E., Berardinelli, A.L., D’Angelo, G., Pini, A., Baranello, G., Mercuri, E.

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S.P.4 Functional changes in Duchenne muscular dystrophy: A 24month longitudinal cohort study von Mazzone, E.S., Pane, M., Scalise, R., Sormani, M.P., Torrente, Y., Berardinelli, A., Messina, S., Cavallaro, F., ′Amico, A. D, Doglio, L., Bruno, C., Politano, L., Frosini, S., Battini, R., Bello, L., Pegoraro, E., Bonfiglio, S., Comi, G., Pini, A., de Sanctis, R., Bianco, F., Vasco, G., Mongini, T., Donati, M.A., Previtali, S., Napolitano, S., Vita, G., Bertini, E., Mercuri, E.

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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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A0998 - A systematic review to evaluate Patient-Reported Outcomes Measures (PROMs) for metastatic prostate cancer according to the COSMIN methodology – A PIONEER wp2 project von Ratti, M.M., Gandaglia, G., Armando, S., Mazzone, E., Scuderi, S., Barletta, F., Mottet, N., Williamson, P.R., Moss, C., Beyer, K., Muhammad Imran, O., Maclennan, S., Zong, J., Cornford, P., Maclennan, S.J., Aiyegbusi, O.L., Van Hemelrijck, M., Alleva, E., Derevianko, A., Sisca, E.S., N’dow, J., Francesco, M., Briganti, A.

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A systematic review to evaluate Patient-Reported Outcomes Measures (PROMs) for metastatic prostate cancer according to the COSMIN methodology – A PIONEER wp2 project von Ratti, M.M., Gandaglia, G., Armando, S., Mazzone, E., Scuderi, S., Barletta, F., Mottet, N., Williamson, P.R., Moss, C., Beyer, K., Muhammad Imran, O., Maclennan, S., Zong, J., Cornford, P., Maclennan, S.J., Aiyegbusi, O.L., Van Hemelrijck, M., Alleva, E., Derevianko, A., Sisca, E.S., N’dow, J., Francesco, M., Briganti, A.

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