Intra-laboratory validation of the Ridascreen® SET Total kit for detecting staphylococcal enterotoxins SEA to SEE in cheese von Ostyn, A, Guillier, F, Prufer, A.-L, Papinaud, I, Messio, S, Krys, S, Lombard, B, Hennekinne, J.-A

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Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial von McDonald, Craig M, Campbell, Craig, Torricelli, Ricardo Erazo, Finkel, Richard S, Flanigan, Kevin M, Goemans, Nathalie, Heydemann, Peter, Kaminska, Anna, Kirschner, Janbernd, Muntoni, Francesco, Osorio, Andrés Nascimento, Schara, Ulrike, Sejersen, Thomas, Shieh, Perry B, Sweeney, H Lee, Topaloglu, Haluk, Tulinius, Már, Vilchez, Juan J, Voit, Thomas, Wong, Brenda, Elfring, Gary, Kroger, Hans, Luo, Xiaohui, McIntosh, Joseph, Ong, Tuyen, Riebling, Peter, Souza, Marcio, Spiegel, Robert J, Peltz, Stuart W, Mercuri, Eugenio, Alfano, Lindsay N, Eagle, Michelle, James, Meredith K, Lowes, Linda, Mayhew, Anna, Mazzone, Elena S, Nelson, Leslie, Rose, Kristy J, Abdel-Hamid, Hoda Z, Apkon, Susan D, Barohn, Richard J, Bertini, Enrico, Bloetzer, Clemens, de Vaud, Lausanne Canton, Butterfield, Russell J, Chabrol, Brigitte, Chae, Jong-Hee, Jongno-gu, Daehak-ro, Comi, Giacomi Pietro, Darras, Basil T, Dastgir, Jahannaz, Desguerre, Isabelle, Escobar, Raul G, Finanger, Erika, Guglieri, Michela, Hughes, Imelda, Iannaccone, Susan T, Jones, Kristi J, Karachunski, Peter, Kudr, Martin, Lotze, Timothy, Mah, Jean K, Mathews, Katherine, Nevo, Yoram, Parsons, Julie, Péréon, Yann, de Queiroz Campos Araujo, Alexandra Prufer, Renfroe, J Ben, de Resende, Maria Bernadete Dutra, Ryan, Monique, Selby, Kathryn, Tennekoon, Gihan, Vita, Giuseppe

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Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled tria... von Nascimento, Andres, Vuillerot, Carole, Boespflug-Tanguy, Odile, Kirschner, Janbernd, Kostera-Pruszczyk, Anna, Kletzl, Heidemarie, Day, John W, Volpe, Joseph J., Posner, John, Daron, Aurore, Delbeke, Patricia, Wittevrongel, Sylvia, De Vos, Elke, De Waele, Liesbeth, Prufer de Queiroz Campos Araujo, Alexandra, Haefeli, Lorena, Chiu, Angela, Oskoui, Maryam, Toffoli, Daniela, Turgeon Desilets, Sarah, Wang, Yi, Shen, Li, Li, Hui, Sang, Tian, Barisic, Nina, Galiot Delic, Martina, Ivkic, Petra Kristina, Kern, Ivana, Momtchilova, Marta Milkova, Milascevic, Darko, Aubertin, Guillaume, Coopman, Stephanie, Debruyne, Philippe, Derlyn, Gilles, Danjoux, Loïc, Deladrière, Élodie, Germa, Virginie, Billaud, Fanny, Le Goff, Lucie, Letellier, Guy, De-Montferrand, Camille, Le-Goff, Laure, Fontaine, Stephanie, Kirschner, Janbernd, Lagréze, Wolf, Bertini, Enrico, D'Amico, Adele, Carlesi, Adelina, Zuffi, Ambra, Comi, Giacomo Pietro, Daniele, Velardo, Minorini, Valeria, Fassini, Federica, Foa, Michaela, Parente, Valeria, Masson, Riccardo, Tremolada, Gemma, Amorelli, Giulia Maria, Orazi, Lorenzo, Coratti, Giorgia, Morishita, Yuko, Goto, Miwako, Tsutsui, Saki, Takatsuji, Yuta, Komaki, Hirofumi, Omori, Miina, Nagura, Hiroe, Usui, Manami, Fudeyasu, Kenishi, Hirata, Kazuhiko, Michiue, Kana, Fujitani, Junko, Seki, Maiko, Fukuyama, Koji, Shibata, Minoru, Kobayashi, Kyoko, Nakamura, Yukie, Sikorski, Adam, Koberda, Mateusz, Stodolska-Koberda, Urszula, Waskowska, Agnieszka, Kolendo, Jagoda, Steinborn, Barbara, Klimas, Monika, Stopa, Marcin, Gajewska, Ewa, Leppenen, Natalya, Brankovic, Vesna, Djokic, Olivera, Planas Pascual, Bernat, Salinas, Marta Peña, Padros, Nuria, Oncel, Ibrahim, Eroglu Ertugru, Nesibe, Kadayifçilar, Sibel, Karaduman, Aynur Ayse, Chiriboga, Claudia, Marquet, Anne, Muelhardt, Nicoletta, Trundell, Dylan

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249P Clinical characteristics of the spinal muscular atrophy patients identified in the Brazilian public health system von Batista, E.C., Zanoteli, E., Ortega, A.B., Junior, M.C.F., Saute, J.A.M., Oliveira, A.S.B., Giannetti, J.G., Pessoa, A.L.S., Prufer, A., Boy, R., Junior, F.G.B., de Albuquerque, C.S.N., Moia, D.D.F., Monfardini, F., Santos, G.P., Soares, R.V.P., Silva, G.S., Rizzo, L.V., Berwanger, O., Fonseca, H.A.R.

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The emerging spectrum of neurodevelopmental comorbidities in early-onset Spinal Muscular Atrophy von Baranello, Giovanni, Roy, Susana Quijano, Servais, Laurent, Munell, Francina, Molinero, Mireia Alvarez, Natera de Benito, Daniel, Nascimento, Andres, Gomez-Andres, David, Comellas, Laura Costa, Exposito, Jessica, Tizzano, Eduardo F., Cuppen, Inge, Van der Pol, Ludo, Aleman, Alberto, Lochmuller, Hanns, McMillan, Hugh, Kirschner, Janbernd, Müller, Cornelia, Oskoui, Maryam, Masson, Riccardo, Bruno, Claudio, Gonorazky, Hernan D., Tesi-Rocha, Carolina, Yaworski, Amanda Marie, Zanoteli, Edmar, Mendonca, Rodrigo, D'Amico, Adele, Cumbo, Francesca, Tosi, Michele, Pane, Marika, Mercuri, Eugenio, Nardes, Flavia, Prufer, Alexandra, Arci, Brenda Klemm, Pascual, Samuel Ignacio, Fattal-Valevski, Aviva, De Waele, Liesbeth, Deconinck, Nicolas, Farrar, Michelle, Haberlova, Jana, Gomez-Garcia de la Banda, Marta, Childs, Anne-Marie, Martos, Cristina, Wraige, Elizabeth, Gowda, Vasantha, Illingworth, Marjorie, Ong, Min, Majundar, Anirban, Hughes, Imelda, Torne, Krupa, Willis, Tracey, Ramdas, Sithara, De Goede, Christian, Erbas, Yasemin, Brusa, Chiara, Scoto, Mariacristina, Muntoni, Francesco

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P212 RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA) von Finkel, R., Farrar, M., Servais, L., Vlodavets, D., Zanoteli, E., Al-Muhaizea, M., Prufer, A., Nelson, L., Fischer, C., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Gaki, E., Fontoura, P., Bertini, E.

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FP.24 RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA) von Finkel, R., Farrar, M., Vlodavets, D., Zanoteli, E., Al-Muhaizea, M., Nelson, L., Prufer, A., Servais, L., Wang, Y., Fisher, C., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Scalco, R., Bertini, E.

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G.P.13.03 Can VLA-4 be applied as a biomarker for prognosis and therapeutic target in Duchenne muscular dystrophy? von Pinto-Mariz, F, Carvalho, L.R, de Mello, W, Prufer, A, Ribeiro, M.G, Alves-Cunha, M.C.S, Riederer, I, Negroni, E, Voit, T, Desguerre, I, Butler-Browne, G, Savino, W, Silva-Barbosa, S.D

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P.362RAINBOWFISH: A study of risdiplam (RG7916) in newborns with pre-symptomatic spinal muscular atrophy (SMA) von Bertini, E., Day, J., Muhaizea, M., Xiong, H., Servais, L., Prufer, A., Tichy, M., Yeung, W., Gorni, K.

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CLINICAL TRIAL HIGHLIGHTS: O.4 RAINBOWFISH: A study of risdiplam in infants with presymptomatic spinal muscular atrophy (SMA) von Servais, L., Al-Muhaizea, M., Farrar, M., Nelson, L., Prufer, A., Finkel, R., Wang, Y., Zanoteli, E., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Scalco, R., Bertini, E.

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CLINICAL TRIAL HIGHLIGHTS von Servais, L., Al-Muhaizea, M., Farrar, M., Nelson, L., Prufer, A., Finkel, R., Wang, Y., Zanoteli, E., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Scalco, R., Bertini, E.

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SMA – THERAPY: P.274 RAINBOWFISH: A study of risdiplam (RG7916) in infants with presymptomatic spinal muscular atrophy (SMA) von Servais, L., Bertini, E., Al-Muhaizea, M., Nelson, L., Prufer, A., Vlodavets, D., Wang, Y., Zanoteli, E., Burke, L., El-Khairi, M., Gorni, K., Kletzl, H., Gerber, M., Scalco, R., Finkel, R.

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SMA – THERAPY von Servais, L., Bertini, E., Al-Muhaizea, M., Nelson, L., Prufer, A., Vlodavets, D., Wang, Y., Zanoteli, E., Burke, L., El-Khairi, M., Gorni, K., Kletzl, H., Gerber, M., Scalco, R., Finkel, R.

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Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy von Shieh, Perry B, Elfring, Gary, Trifillis, Panayiota, Santos, Claudio, Peltz, Stuart W, Parsons, Julie A, Apkon, Susan, Darras, Basil T, Campbell, Craig, McDonald, Craig M

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SMA THERAPIES II AND BIOMARKERS: P.256A study of RG7916 in infants with pre-symptomatic spinal muscular atrophy von Bertini, E., Day, J., Al Muhaizea, M., Xiong, H., Servais, L., Prufer, A., Buchbjerg, J., Armstrong, G., Gorni, K., Khwaja, O.

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SMA THERAPIES II AND BIOMARKERS von Bertini, E., Day, J., Al Muhaizea, M., Xiong, H., Servais, L., Prufer, A., Buchbjerg, J., Armstrong, G., Gorni, K., Khwaja, O.

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P16.13 Genetic test for DMD and SMA may be guided by simple clinical features von Araujo, A. Prufer de Q.C., Ramos, V.G., Cabello, P.H.

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Intra-laboratory validation of the Ridascreen® SET Total kit for detecting staphylococcal enterotoxins SEA to SEE in cheese: SEA to SEE detection in cheeses von Ostyn, A., Guillier, F., Prufer, A.-L., Papinaud, I., Messio, S., Krys, S., Lombard, B., Hennekinne, J.-A.

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Brain Activation by Peptide Pro-Leu-Gly-NH2 (MIF-1) von Khan, Reas S., Yu, Chuanhui, Kastin, Abba J., He, Yi, Ehrensing, Rudolph H., Hsuchou, Hung, Stone, Kirsten Prufer, Pan, Weihong

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426P Ankle foot orthosis prescription for Duchenne muscular dystrophy patients: a retrospective study von Prufer De, Q.C. Araujo A, Rebel, M. Ferreira, Pereira, J. Almeida, Brasil, L. Mello, Pereira, A. Chacon, Kovacs, A. Horokoski, Fortes, C. Pereira Dias Drumond, dos Santos, F. Nardes, Landgraaf, J. de Fatima, Stajnbock, F.

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