Chiari type I malformation causing central apnoeas in a 4-month-old boy von Van den Broek, Mieke J.A, Arbues, Anne-Sophie, Chalard, François, Pinto Da Costa, Nathalia M, Trang, Ha, Dorfmuller, Georg, Evrard, Philippe P, Husson, Isabelle, Servais, Laurent J

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Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy von Feraudy, Yvan, Ben Yaou, Rabah, Wahbi, Karim, Stalens, Caroline, Stantzou, Amalia, Laugel, Vincent, Desguerre, Isabelle, Servais, Laurent, Leturcq, France, Amthor, Helge

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Anterior spinal artery syndrome after aortic surgery in a child von Servais, Laurent J, Rivelli, Sarah K, Dachy, Bernard A, Christophe, Catherine D, Dan, Bernard

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Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study von Voit, Thomas, Prof, Topaloglu, Haluk, Prof, Straub, Volker, Prof, Muntoni, Francesco, Prof, Deconinck, Nicolas, MD, Campion, Giles, MD, De Kimpe, Sjef J, PhD, Eagle, Michelle, PhD, Guglieri, Michela, MD, Hood, Steve, PhD, Liefaard, Lia, PhD, Lourbakos, Afrodite, PhD, Morgan, Allison, BSc, Nakielny, Joanna, MB, Quarcoo, Naashika, PhD, Ricotti, Valeria, MB, Rolfe, Katie, MSc, Servais, Laurent, MD, Wardell, Claire, DPhil, Wilson, Rosamund, PhD, Wright, Padraig, MD, Kraus, John E, MD

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Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients von Jeanson-Leh, Laurence, Lameth, Julie, Krimi, Soraya, Buisset, Julien, Amor, Fatima, Le Guiner, Caroline, Barthélémy, Inès, Servais, Laurent, Blot, Stéphane, Voit, Thomas, Israeli, David

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Risdiplam in Type 1 Spinal Muscular Atrophy von Baranello, Giovanni, Darras, Basil T, Day, John W, Deconinck, Nicolas, Klein, Andrea, Masson, Riccardo, Mercuri, Eugenio, Rose, Kristy, El-Khairi, Muna, Gerber, Marianne, Gorni, Ksenija, Khwaja, Omar, Kletzl, Heidemarie, Scalco, Renata S, Seabrook, Timothy, Fontoura, Paulo, Servais, Laurent

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Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy von Frank, Diane E., Schnell, Frederick J., Akana, Cody, El-Husayni, Saleh H., Desjardins, Cody A., Morgan, Jennifer, Charleston, Jay S., Sardone, Valentina, Domingos, Joana, Dickson, George, Straub, Volker, Guglieri, Michela, Mercuri, Eugenio, Servais, Laurent, Muntoni, Francesco

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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy von Finkel, Richard S, Mercuri, Eugenio, Darras, Basil T, Connolly, Anne M, Kuntz, Nancy L, Kirschner, Janbernd, Chiriboga, Claudia A, Saito, Kayoko, Servais, Laurent, Tizzano, Eduardo, Topaloglu, Haluk, Tulinius, Már, Montes, Jacqueline, Glanzman, Allan M, Bishop, Kathie, Zhong, Z. John, Gheuens, Sarah, Bennett, C. Frank, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C

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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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An integrated modelling methodology for estimating the prevalence of centronuclear myopathy von Vandersmissen, I., Biancalana, V., Servais, L., Dowling, J.J., Vander Stichele, G., Van Rooijen, S., Thielemans, L.

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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial von Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

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Capillary Electrophoresis-Mass Spectrometry at Trial by Metabo-Ring: Effective Electrophoretic Mobility for Reproducible and Robust Compound Annotation von Drouin, Nicolas, van Mever, Marlien, Zhang, Wei, Tobolkina, Elena, Ferre, Sabrina, Servais, Anne-Catherine, Gou, Marie-Jia, Nyssen, Laurent, Fillet, Marianne, Lageveen-Kammeijer, Guinevere S. M., Nouta, Jan, Chetwynd, Andrew J., Lynch, Iseult, Thorn, James A., Meixner, Jens, Lossner, Christopher, Taverna, Myriam, Liu, Sylvie, Tran, N. Thuy, Francois, Yannis, Lechner, Antony, Nehm, Reine, Banni, Ghassan Al Hamoui Dit, Nasreddine, Rouba, Colas, Cyril, Lindner, Herbert H., Faserl, Klaus, Neusuess, Christian, Nelke, Manuel, Laemmerer, Stefan, Perrin, Catherine, Bich-Muracciole, Claudia, Barbas, Coral, Lopez Gonzalvez, Angeles, Guttman, Andras, Szigeti, Marton, Britz-McKibbin, Philip, Kroezen, Zachary, Shanmuganathan, Meera, Nemes, Peter, Portero, Erika P., Hankemeier, Thomas, Codesido, Santiago, Gonzalez-Ruiz, Victor, Rudaz, Serge, Ramautar, Rawi

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Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study von Ricotti, V., Selby, V., Ridout, D., Domingos, J., Decostre, V., Mayhew, A., Eagle, M., Butler, J., Guglieri, M., Van der Holst, M., Jansen, M., Verschuuren, J.J.G.M., de Groot, I.J.M., Niks, E.H., Servais, L., Straub, V., Voit, T., Hogrel, J.Y., Muntoni, F.

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Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study von Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Done, Nicolae, Yao, Zhiwen, Goemans, Nathalie, McDonald, Craig, Mercuri, Eugenio, Niks, Erik H, Wong, Brenda, Vandenborne, Krista, Straub, Volker, de Groot, Imelda J M, Tian, Cuixia, Manzur, Adnan, Dieye, Ibrahima, Lane, Henry, Ward, Susan J, Servais, Laurent

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Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial von Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

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Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy von Servais, Laurent, Lair, Lindsey Lee, Connolly, Anne M, Byrne, Barry J, Chen, Karen S, Coric, Vlad, Qureshi, Irfan, Durham, Susan, Campbell, Daniel J, Maclaine, Grant, Marin, Jackie, Bechtold, Clifford

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European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy von Kirschner, Janbernd, Butoianu, Nina, Goemans, Nathalie, Haberlova, Jana, Kostera-Pruszczyk, Anna, Mercuri, Eugenio, van der Pol, W. Ludo, Quijano-Roy, Susana, Sejersen, Thomas, Tizzano, Eduardo F., Ziegler, Andreas, Servais, Laurent, Muntoni, Francesco

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Crowdfunding for neuromuscular disease treatment: the ethical implications von Livingstone, Angus, Servais, Laurent, Wilkinson, Dominic J C

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The ethics of crowdfunding in paediatric neurology von Livingstone, Angus, Servais, Laurent, Wilkinson, Dominic J. C.

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Childhood-onset form of myotonic dystrophy type 1 and autism spectrum disorder: Is there comorbidity? von Angeard, N., Huerta, E., Jacquette, A., Cohen, D., Xavier, J., Gargiulo, M., Servais, L., Eymard, B., Héron, D.

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