Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium von Boemer, François, Caberg, Jean-Hubert, Beckers, Pablo, Dideberg, Vinciane, di Fiore, Samantha, Bours, Vincent, Marie, Sandrine, Dewulf, Joseph, Marcelis, Lionel, Deconinck, Nicolas, Daron, Aurore, Blasco-Perez, Laura, Tizzano, Eduardo, Hiligsmann, Mickaël, Lombet, Jacques, Pereira, Tatiana, Lopez-Granados, Lucia, Shalchian-Tehran, Sarvnaz, van Assche, Véronique, Willems, Arabelle, Huybrechts, Sofie, Mast, Bénédicte, van Olden, Rudolf, Dangouloff, Tamara, Servais, Laurent

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Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study von Chabanon, Aurélie, Seferian, Andreea Mihaela, Daron, Aurore, Péréon, Yann, Cances, Claude, Vuillerot, Carole, De Waele, Liesbeth, Cuisset, Jean-Marie, Laugel, Vincent, Schara, Ulrike, Gidaro, Teresa, Gilabert, Stéphanie, Hogrel, Jean-Yves, Baudin, Pierre-Yves, Carlier, Pierre, Fournier, Emmanuel, Lowes, Linda Pax, Hellbach, Nicole, Seabrook, Timothy, Toledano, Elie, Annoussamy, Mélanie, Servais, Laurent

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Normative data on spontaneous stride velocity, stride length, and walking activity in a non-controlled environment von Poleur, Margaux, Ulinici, Ana, Daron, Aurore, Schneider, Olivier, Farra, Fabian Dal, Demonceau, Marie, Annoussamy, Mélanie, Vissière, David, Eggenspieler, Damien, Servais, Laurent

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Natural history of Type 2 and 3 spinal muscular atrophy: 2‐year NatHis‐SMA study von Annoussamy, Mélanie, Seferian, Andreea M., Daron, Aurore, Péréon, Yann, Cances, Claude, Vuillerot, Carole, De Waele, Liesbeth, Laugel, Vincent, Schara, Ulrike, Gidaro, Teresa, Lilien, Charlotte, Hogrel, Jean‐Yves, Carlier, Pierre, Fournier, Emmanuel, Lowes, Linda, Gorni, Ksenija, Ly‐Le Moal, Myriam, Hellbach, Nicole, Seabrook, Timothy, Czech, Christian, Hermosilla, Ricardo, Servais, Laurent

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Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression von Monseur, Arnaud, Carlin, Bradley P., Boulanger, Bruno, Seferian, Andreea, Servais, Laurent, Freitag, Chris, Thielemans, Leen

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Nusinersen in patients older than 7 months with spinal muscular atrophy type 1 von Aragon-Gawinska, Karolina, Seferian, Andreea, Daron, Aurore, Gargaun, Elena, Vuillerot, Carole, Cances, Claude, Ropars, Juliette, Chouchane, Mondher, Cuppen, Inge, Hughes, Imelda, Illingworth, Marjorie, Marini-Bettolo, Chiara, Rambaud, Jerome, Taytard, Jessica, Annoussamy, Melanie, Scoto, Mariacristina, Gidaro, Teresa, Servais, Laurent

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Case report of cerebral cystic echinococcosis in a 5-year-old child von Menschaert, Denis, Daron, Aurore, Frere, Julie

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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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Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort study von Aragon-Gawinska, Karolina, Seferian, Andreea M, Daron, Aurore, Gargaun, Elena, Vuillerot, Carole, Cances, Claude, Ropars, Juliette, Chouchane, Mondher, Cuppen, Inge, Hughes, Imelda, Illingworth, Marjorie, Marini-Bettolo, Chiara, Rambaud, Jerome, Taytard, Jessica, Annoussamy, Melanie, Scoto, Mariacristina, Gidaro, Teresa, Servais, Laurent

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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial von Mercuri, Eugenio, Muntoni, Francesco, Baranello, Giovanni, Masson, Riccardo, Boespflug-Tanguy, Odile, Bruno, Claudio, Corti, Stefania, Daron, Aurore, Deconinck, Nicolas, Servais, Laurent, Straub, Volker, Ouyang, Haojun, Chand, Deepa, Tauscher-Wisniewski, Sitra, Mendonca, Nuno, Lavrov, Arseniy, Seferian, A., De Lucia, S., Tachibana, S., Jollet, A., Mouffak, S., Pedemonte, M., Brolatti, N., Morando, S., Vanlander, A., De Vos, E., Tahon, V., Govoni, A., Magri, F., Comi, G., Foa, M., Parente, V., Buscemi, L., Dal Farra, F., Schneider, O., Jonas, A., Defeldre, A.C., Pagliano, E., Zanin, R., Arnoldi, M.T., Schembri, V., Del Sole, M., Mandelli, A., Pera, M.C., Antonaci, L., Coratti, G., de Sanctis, R., Pane, M., Scoto, M., Groves, K., Edel, L., Abel, F., Van Ruiten, H., Lofra, R.M., Thompson, E.

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Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium von Dangouloff, Tamara, Thokala, Praveen, Stevenson, Matthew D, Deconinck, Nicolas, D'Amico, Adèle, Daron, Aurore, Delstanche, Stephanie, Servais, Laurent, Hiligsmann, Mickael

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X-linked myotubular myopathy: A prospective international natural history study von Annoussamy, Mélanie, Lilien, Charlotte, Gidaro, Teresa, Gargaun, Elena, Chê, Virginie, Schara, Ulrike, Gangfuß, Andrea, DʼAmico, Adele, Dowling, James J, Darras, Basil T, Daron, Aurore, Hernandez, Arturo, de Lattre, Capucine, Arnal, Jean-Michel, Mayer, Michèle, Cuisset, Jean-Marie, Vuillerot, Carole, Fontaine, Stéphanie, Bellance, Rémi, Biancalana, Valérie, Buj-Bello, Ana, Hogrel, Jean-Yves, Landy, Hal, Servais, Laurent

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Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen von Aragon‐Gawinska, Karolina, Daron, Aurore, Ulinici, Ana, Vanden Brande, Laura, Seferian, Andreea, Gidaro, Teresa, Scoto, Mariacristina, Deconinck, Nicolas, Servais, Laurent, Benezit, Audrey, Mathieu, Marie‐Laure, Cances, Claude, Durigneux, Julien, Ropars, Juliette, Chouchane, Mondher, Forey, Peggy, Lazaro, Leila, Hughes, Imelda, Illingworth, Marjorie, Marini‐Bettolo, Chiara, Cuppen, Inge, Modrzejewska, Sandra, Balintova, Zdenka, Haberlova, Jana, Drimtzia, Kate, Blaschek, Astrid, Ambegankoar, Gautam, Annoussamy, Melanie

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Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy von Fouarge, Eve, Monseur, Arnaud, Boulanger, Bruno, Annoussamy, Mélanie, Seferian, Andreea M, De Lucia, Silvana, Lilien, Charlotte, Thielemans, Leen, Paradis, Khazal, Cowling, Belinda S, Freitag, Chris, Carlin, Bradley P, Servais, Laurent

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Scoring People With Spinal Muscular Atrophy on the Motor Function Measure Using the Microsoft Kinect von Vincent-Genod, Dominique, Rippert, Pascal, Coton, Justine, Le Goff, Laure, Barriere, Aurélie, Berruyer, Anne, Bernard, Marjorie, Garde, Camille, Gutierrez-Garcia, Marta, Gilabert, Stéphanie, Gomes-Lisboade-Souza, Adriana, Daron, Aurore, Servais, Laurent, Thomann, Guillaume, Vuillerot, Carole

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Scoring People With Spinal Muscular Atrophy on the Motor Function Measure Using the Microsoft Kinect von Vincent-Genod, Dominique, Rippert, Pascal, Coton, Justine, Le Goff, Laure, Barriere, Aurélie, Berruyer, Anne, Bernard, Marjorie, Garde, Camille, Gutierrez-Garcia, Marta, Gilabert, Stéphanie, Gomes-Lisboade-Souza, Adriana, Daron, Aurore, Servais, Laurent, Thomann, Guillaume, Vuillerot, Carole

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Natural History of Type 2 and 3 Spinal Muscular Atrophy (SMA): Longitudinal 2-year NatHis-SMA Study (530) von Servais, Laurent, Seferian, Andreea Mihaela, Daron, Aurore, Péréon, Yann, Cances, Claude, Vuillerot, Carole, De Waele, Liesbeth MH, Laugel, Vincent, Schara, Ulrike, Gidaro, Teresa, Lilien, Charlotte, Hogrel, Jean-Yves, Baudin, Pierre-Yves, Carlier, Pierre, Fournier, Emmanuel, Lowes, Linda Pax, Gorni, Ksenija, Moal, Myriam Ly-Le, Hellbach, Nicole, Seabrook, Timothy, Czech, Christian, Hermosilla, Ricardo, Annoussamy, Mélanie

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A novel CACNA1A mutation results in episodic ataxia with migrainous features without headache von Magis, Delphine, Boon, Elles, Coppola, Gianluca, Daron, Aurore, Schoenen, Jean

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X-linked myotubular myopathy von Annoussamy, Mélanie, Lilien, Charlotte, Gidaro, Teresa, Gargaun, Elena, Chê, Virginie, Schara, Ulrike, Gangfuß, Andrea, d'Amico, Adele, Dowling, James, Darras, Basil, Daron, Aurore, Hernandez, Arturo, de Lattre, Capucine, Arnal, Jean-Michel, Mayer, Michèle, Cuisset, Jean-Marie, Vuillerot, Carole, Fontaine, Stéphanie, Bellance, Rémi, Biancalana, Valérie, Buj-Bello, Ana, Hogrel, Jean-Yves, Landy, Hal, Servais, Laurent

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Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study von Chabanon, Aurelie, Seferian, Andreea Mihaela, Daron, Aurore, Pereon, Yann, Cances, Claude, Vuillerot, Carole, De Waele, Liesbeth, Cuisset, Jean-Marie, Laugel, Vincent, Schara, Ulrike, Gidaro, Teresa, Gilabert, Stephanie, Hogrel, Jean-Yves, Baudin, Pierre-Yves, Carlier, Pierre, Fournier, Emmanuel, Lowes, Linda Pax, Hellbach, Nicole, Seabrook, Timothy, Toledano, Elie, Annoussamy, Melanie, Servais, Laurent

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