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... by 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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Early and long-term effect of the treatment with pyridostigmine in patients with GMPPB-related congenital myasthenic syndrome by Bobadilla-Quesada, Edna Julieth, Natera-de Benito, Daniel, Carrera-García, Laura, Ortez, Carlos, Exposito-Escudero, Jessica, Jimenez-Mallebrera, Cecilia, Jou, Cristina, Codina, Anna, Corbera, Joan, Moya, Obdulia, Saez, Veronica, Gonzalez-Quereda, Lidia, Gallano, Pia, Colomer, Jaume, Cuadras, Daniel, Medina, Julita, Yoldi, María Eugenia, Nascimento, Andrés

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The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy by Muntoni, Francesco, Byrne, Barry J., McMillan, Hugh J., Ryan, Monique M., Wong, Brenda L., Dukart, Juergen, Bansal, Amita, Cosson, Valerie, Dreghici, Roxana, Guridi, Maitea, Rabbia, Michael, Staunton, Hannah, Tirucherai, Giridhar S., Yen, Karl, Yuan, Xiling, Wagner, Kathryn R.

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Complex regional pain syndrome of the face in a child by González‐Cantero, Álvaro, Sánchez‐Moya, Ana Isabel, Pérez‐Hortet, Cristina, Martinez‐Lorenzo, Elena, Gómez‐Dorado, Blas, Molina‐Figuera, Elvira, García‐Olmedo, Obdulia, Schoendorff‐Ortega, Cristina, Sherry, David D.

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Successful Treatment of Refractory Type 1 Pityriasis Rubra Pilaris With Apremilast by Molina-Figuera, Elvira, González-Cantero, Álvaro, Martínez-Lorenzo, Elena, Sánchez-Moya, Ana-Isabel, García-Olmedo, Obdulia, Gómez-Dorado, Blas, Schoendorff-Ortega, Cristina

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Successful Treatment of Refractory Type 1 Pityriasis Rubra Pilaris With Apremilast by Molina-Figuera, Elvira, González-Cantero, Álvaro, Martínez-Lorenzo, Elena, Sánchez-Moya, Ana-Isabel, García-Olmedo, Obdulia, Gómez-Dorado, Blas, Schoendorff-Ortega, Cristina

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