"Be an ambassador for change that you would like to see": a call to action to all stakeholders for co-creation in healthcare and medical research to improve quality of life of peop... von Ambrosini, Anna, Quinlivan, Ros, Sansone, Valeria A, Meijer, Ingeborg, Schrijvers, Guus, Tibben, Aad, Padberg, George, de Wit, Maarten, Sterrenburg, Ellen, Mejat, Alexandre, Breukel, Alexandra, Rataj, Michal, Lochmueller, Hanns, Willmann, Raffaella, 235th ENMC workshop study group

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Approximation, Randomization, and Combinatorial Optimization: Algorithms and Techniques: 4th International Workshop on Approximation Algorithms for Combinatorial Optimization Probl... von Goemans, Michel, Jansen, Klaus, Rolim, José D. P, Trevisan, Luca

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1st Workshop on Upper-Extremity Assistive Technology for People with Duchenne: State of the art, emerging avenues, and challenges: April 27th 2015, London, United Kingdom von Bergsma, Arjen, Lobo-Prat, Joan, Vroom, Elizabeth, Furlong, Pat, Herder, Just L

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How robust is ACTIVLIM for the follow-up of activity limitations in patients with neuromuscular diseases? von Batcho, Charles Sèbiyo, Van den Bergh, Peter Y.K, Van Damme, Philip, Roy, Anna J, Thonnard, Jean-Louis, Penta, Massimo, BNMDR Scientific Committee

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Neuromuscular Disorders in Children: A Multidisciplinary Approach to Management von Deconinck, Nicolas

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Early stages of building a rare disease registry, methods and 2010 data from the Belgian Neuromuscular Disease Registry (BNMDR) von Roy, Anna J, Van den Bergh, Peter, Van Damme, Philip, Doggen, Kris, Van Casteren, Viviane, The BNMDR Scientific Committee

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A Stochastic Probing Problem with Applications von Gupta, Anupam, Nagarajan, Viswanath

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Ripples in scalp EEGs of children: co-occurrence with sleep-specific transients and occurrence across sleep stages von Mooij, Anne H, Frauscher, Birgit, Goemans, Sophie A M, Huiskamp, Geertjan J M, Braun, Kees P J, Zijlmans, Maeike

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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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Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial von Bertini, Enrico, Prof, Dessaud, Eric, PhD, Mercuri, Eugenio, Prof, Muntoni, Francesco, Prof, Kirschner, Janbernd, Prof, Reid, Carol, PhD, Lusakowska, Anna, MD, Comi, Giacomo P, Prof, Cuisset, Jean-Marie, MD, Abitbol, Jean-Louis, MD, Scherrer, Bruno, PhD, Ducray, Patricia Sanwald, PhD, Buchbjerg, Jeppe, MSc, Vianna, Eduardo, PhD, van der Pol, W Ludo, MD, Vuillerot, Carole, MD, Blaettler, Thomas, MD, Fontoura, Paulo, MD

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Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go von Dangouloff, Tamara, Vrščaj, Eva, Servais, Laurent, Osredkar, Damjan, Adoukonou, Thierry, Aryani, Omid, Barisic, Nina, Bashiri, Fahad, Bastaki, Laila, Benitto, Afaf, Omran, Tawfeg Ben, Bernert, Guenther, Bertini, Enrico, Borde, Patricia, Born, Peter, Boustani, Rose-Mary, Butoianu, Nina, Castiglioni, Claudia, Catibusic, Feriha, Chan, Sophelia, Chien, Yin Hsiu, Christodoulou, Kyproula, Dejsuphong, Donniphat, Farrar, Michelle, Filip, Duma, Goemans, Nathalie, Guinhouya, Kokou, Haberlova, Jana, Hadzsiev, Kinga, Hovhannesyan, Kristine, Isohanni, Pirjo, Radovic, Nelica Ivanovic, Jacquier, David, Jalloh, Alusine, Jedrzejowska, Maria, Kandawasvika, Gwen, Kaputu, Celestin, Kawatu, Nfwama, Kernohan, Kristin, Kirschner, Jan, Klink, Barbara, Kodsy, Sherry, Kouame-Assouan, Ange-Eric, Kravljanac, Ruzica, Kreile, Madara, Litvinenko, Ivan, McMillan, Hugh, Mesa, Sandra, Mohamed, Inaam, Kanzoska, Liljana Muaremoska, Nevo, Yoram, Nguefack, Seraphin, Nkole, Kafula, O'Grady, Gina, O'Rourke, Declan, Oskoui, Maryam, Piazzon, Flavia, Poddighe, Dimitri, Prasauskiene, Audrone, Prieto, Juan, Rasmussen, Magnhild, Razafindrasata, Santara, Saha, Narayan, Saito, Kayoko, Sakadi, Foksouna, Sangare, Modibo, Schroth, Mary, Shalkevich, Leanid, Shatillo, Andriy, Suthar, Renu, Szabo, Lena, Tatishvili, Nana, Tazir, Meriem, Tizzano, Eduardo, Topaloglu, Haluk, Tulinius, Mar, van der Pol, Ludo, Vazquez, Gabriel, Vlodavets, Dimitry, Wanigasinghe, Jithangi, Wilmshurst, Jo, Xiong, Hui, Zafeiriou, Dimitrios, Zamba, Eleni

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Ataluren treatment of patients with nonsense mutation dystrophinopathy von Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo P., Connolly, Anne M., Day, John W., Flanigan, Kevin M., Goemans, Nathalie, Jones, Kristi J., Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James B., Russman, Barry, Ryan, Monique M., Tulinius, Mar, Voit, Thomas, Moore, Steven A., Lee Sweeney, H., Abresch, Richard T., Coleman, Kim L., Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan M., Henricson, Erik, Barth, Jay, Elfring, Gary L., Reha, Allen, Spiegel, Robert J., O'donnell, Michael W., Peltz, Stuart W., Mcdonald, Craig M.

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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management von Bushby, Katharine, MD, Finkel, Richard, MD, Birnkrant, David J, MD, Case, Laura E, DPT, Clemens, Paula R, MD, Cripe, Linda, MD, Kaul, Ajay, MD, Kinnett, Kathi, MSN, McDonald, Craig, MD, Pandya, Shree, PT, Poysky, James, PhD, Shapiro, Frederic, MD, Tomezsko, Jean, PhD, Constantin, Carolyn, PhD

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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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Diagnostic approach to the congenital muscular dystrophies von Bönnemann, Carsten G, Wang, Ching H, Quijano-Roy, Susana, Deconinck, Nicolas, Bertini, Enrico, Ferreiro, Ana, Muntoni, Francesco, Sewry, Caroline, Béroud, Christophe, Mathews, Katherine D, Moore, Steven A, Bellini, Jonathan, Rutkowski, Anne, North, Kathryn N

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International teachers for tomorrow's school. Opportunities and challenges of the professional re-entry of international teachers in selected European countries

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A joint international consensus statement for measuring quality of survival for patients with childhood cancer von van Kalsbeek, Rebecca J., Hudson, Melissa M., Mulder, Renée L., Ehrhardt, Matthew, Green, Daniel M., Mulrooney, Daniel A., Hakkert, Jessica, den Hartogh, Jaap, Nijenhuis, Anouk, van Santen, Hanneke M., Schouten-van Meeteren, Antoinette Y. N., van Tinteren, Harm, Verbruggen, Lisanne C., Conklin, Heather M., Jacola, Lisa M., Webster, Rachel Tillery, Partanen, Marita, Kollen, Wouter J. W., Grootenhuis, Martha A., Pieters, Rob, Kremer, Leontien C. M.

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A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy von Victor, Ronald G, Sweeney, H Lee, Finkel, Richard, McDonald, Craig M, Byrne, Barry, Eagle, Michelle, Goemans, Nathalie, Vandenborne, Krista, Dubrovsky, Alberto L, Topaloglu, Haluk, Miceli, M Carrie, Furlong, Pat, Landry, John, Elashoff, Robert, Cox, David

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269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands von Naarding, Karin J., Stimpson, Georgia, Ward, Susan J., Goemans, Nathalie, McDonald, Craig, Mercuri, Eugenio, Muntoni, Francesco, Goemans, Nathalie, McDonald, Craig, Mercuri, Eugenio, Muntoni , Francesco, Ward, Susan J., Bettica, Paolo, Binks, Michael, Duong, Tina, Ferreiro, Ana, Furlong, Patricia, Guglieri, Michela, Hendrix, Suzanne, Henricson, Erik, Hogrel, Jean-Yves, Klein, Matthew, Leffler, Mindy, Martinez, Terina, Naarding, Karin J., Niks, Erik, Servais, Laurent, Signorovitch, James, Stimpson , Georgia, Van Ieperen, Frank, Vroom, Elizabeth

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