The First Decade of Journal of Neuromuscular Diseases: Supporting and Advancing the Rapidly Evolving Field of Translational Research von Lochmüller, Hanns, Bönnemann, Carsten G.

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081 IgLON5 autoimmunity in two cases with peripheral nervous system features von Rudaks, Laura I, Fung, Victor SC, Halpern, Jean-Pierre, Ahmad, Omar

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Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care von Schorling, David C., Pechmann, Astrid, Kirschner, Janbernd

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Current Classification and Management of Inflammatory Myopathies von Schmidt, Jens

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Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 – A Prospective Observational Study von Walter, Maggie C., Wenninger, Stephan, Thiele, Simone, Stauber, Julia, Hiebeler, Miriam, Greckl, Eva, Stahl, Kristina, Pechmann, Astrid, Lochmüller, Hanns, Kirschner, Janbernd, Schoser, Benedikt

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Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening von Glascock, Jacqueline, Sampson, Jacinda, Haidet-Phillips, Amanda, Connolly, Anne, Darras, Basil, Day, John, Finkel, Richard, Howell, R. Rodney, Klinger, Katherine, Kuntz, Nancy, Prior, Thomas, Shieh, Perry B., Crawford, Thomas O., Kerr, Douglas, Jarecki, Jill

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Revised Recommendations for the Treatment of Infants Diagnosed with Spinal Muscular Atrophy Via Newborn Screening Who Have 4 Copies of SMN2 von Glascock, Jacqueline, Sampson, Jacinda, Connolly, Anne M., Darras, Basil T., Day, John W., Finkel, Richard, Howell, R. Rodney, Klinger, Katherine W., Kuntz, Nancy, Prior, Thomas, Shieh, Perry B., Crawford, Thomas O., Kerr, Douglas, Jarecki, Jill

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Gene Therapy for Duchenne Muscular Dystrophy von Elangkovan, Nertiyan, Dickson, George

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E-Health & Innovation to Overcome Barriers in Neuromuscular Diseases. Report from the 3rd eNMD Congress: Pisa, Italy, 29–30 October 2021: Remote Monitoring: New Solutions for New Avenues in Neuromuscular Disorders von Schirinzi, Erika, Bochicchio, Mario Alessandro, Lochmüller, Hanns, Vissing, John, Jordie-Diaz-Manerae, Evangelista, Teresinha, Plançon, Jean-Philippe, Fanucci, Luca, Marini, Marco, Tonacci, Alessandro, Mancuso, Michelangelo, Segovia-Kueny, Sandrine, Toscano, Antonio, Angelini, Corrado, Schoser, Benedikt, Sacconi, Sabrina, Siciliano, Gabriele

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AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort von Al-Zaidy, Samiah A., Kolb, Stephen J., Lowes, Linda, Alfano, Lindsay N., Shell, Richard, Church, Kathleen R., Nagendran, Sukumar, Sproule, Douglas M., Feltner, Douglas E., Wells, Courtney, Ogrinc, Francis, Menier, Melissa, L’Italien, James, Arnold, W. David, Kissel, John T., Kaspar, Brian K., Mendell, Jerry R.

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Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database von Koeks, Zaïda, Bladen, Catherine L., Salgado, David, van Zwet, Erik, Pogoryelova, Oksana, McMacken, Grace, Monges, Soledad, Foncuberta, Maria E., Kekou, Kyriaki, Kosma, Konstantina, Dawkins, Hugh, Lamont, Leanne, Bellgard, Matthew I., Roy, Anna J., Chamova, Teodora, Guergueltcheva, Velina, Chan, Sophelia, Korngut, Lawrence, Campbell, Craig, Dai, Yi, Wang, Jen, Barišić, Nina, Brabec, Petr, Lähdetie, Jaana, Walter, Maggie C., Schreiber-Katz, Olivia, Karcagi, Veronika, Garami, Marta, Herczegfalvi, Agnes, Viswanathan, Venkatarman, Bayat, Farhad, Buccella, Filippo, Ferlini, Alessandra, Kimura, En, van den Bergen, Janneke C., Rodrigues, Miriam, Roxburgh, Richard, Lusakowska, Anna, Kostera-Pruszczyk, Anna, Santos, Rosário, Neagu, Elena, Artemieva, Svetlana, Rasic, Vedrana Milic, Vojinovic, Dina, Posada, Manuel, Bloetzer, Clemens, Klein, Andrea, Díaz-Manera, Jordi, Gallardo, Eduard, Karaduman, A. Ayşe, Oznur, Tunca, Topaloğlu, Haluk, El Sherif, Rasha, Stringer, Angela, Shatillo, Andriy V., Martin, Ann S., Peay, Holly L., Kirschner, Jan, Flanigan, Kevin M., Straub, Volker, Bushby, Kate, Béroud, Christophe, Verschuuren, Jan J., Lochmüller, Hanns

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One Year of Newborn Screening for SMA – Results of a German Pilot Project von Vill, Katharina, Kölbel, Heike, Schwartz, Oliver, Blaschek, Astrid, Olgemöller, Bernhard, Harms, Erik, Burggraf, Siegfried, Röschinger, Wulf, Durner, Jürgen, Gläser, Dieter, Nennstiel, Uta, Wirth, Brunhilde, Schara, Ulrike, Jensen, Beate, Becker, Marc, Hohenfellner, Katharina, Müller-Felber, Wolfgang

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Molecular Mechanisms of Skeletal Muscle Hypertrophy von Schiaffino, Stefano, Reggiani, Carlo, Akimoto, Takayuki, Blaauw, Bert

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Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment1 von McGrattan, Katlyn E., Shell, Richard D., Hurst-Davis, Rebecca, Young, Sally Dunaway, O’Brien, Eamonn, Lavrov, Arseniy, Wallach, Shiri, LaMarca, Nicole, Reyna, Sandra P., Darras, Basil T.

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Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials von Carlier, Pierre G., Marty, Benjamin, Scheidegger, Olivier, Loureiro de Sousa, Paulo, Baudin, Pierre-Yves, Snezhko, Eduard, Vlodavets, Dmitry

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Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany von Pechmann, Astrid, Langer, Thorsten, Schorling, David, Stein, Sabine, Vogt, Sibylle, Schara, Ulrike, Kölbel, Heike, Schwartz, Oliver, Hahn, Andreas, Giese, Kerstin, Johannsen, Jessika, Denecke, Jonas, Weiß, Claudia, Theophil, Manuela, Kirschner, Janbernd

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Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1 von McDonald, Craig M, Mayer, Oscar H, Hor, Kan N, Miller, Debra, Goemans, Nathalie, Henricson, Erik K, Marden, Jessica R, Freimark, Jonathan, Lane, Henry, Zhang, Adina, Frean, Molly, Trifillis, Panayiota, Koladicz, Karyn, Signorovitch, James

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Telemedicine in Neuromuscular Diseases During Covid-19 Pandemic: ERN-NMD European Survey von El-Hassar, Lynda, Amara, Ahmed, Sanson, Benoit, Lacatus, Oana, Belhouchet, Ahmed Amir, Kroneman, Madelon, Claeys, Kristl, Plancon, Jean Philippe, Rodolico, Carmelo, Primiano, Guido, Trojsi, Francesca, Filosto, Massimiliano, Mongini, Tiziana Enrica, Bortolani, Sara, Monforte, Mauro, Carraro, Elena, Maggi, Lorenzo, Ricci, Federica, Silani, Vincenzo, Orsucci, Daniele, Creange, Alain, Pereon, Yann, Stojkovic, Tanya, van der Beek, Nadine Anna Maria Elisabeth, Toscano, Antonio, Pareyson, Davide, Attarian, Shahram, Van den Bergh, Peter Y.K, Remiche, Gauthier, Hoeijmakers, Janneke G.J, Badrising, Umesh, Voermans, Nicol C, Kaindl, Angela M, Schara-Schmidt, Ulrike, Schoser, Benedikt, Gazzerro, Elisabetta, Haberlova, Jana, Vohanka, Stanislav, Pal, Endre, Molnar, Maria Judit, Leonardis, Lea, Tournev, Ivailo L, Osorio, Andres Nascimento, Olive, Montse, Muelas, Nuria, Alonso-Perez, Jorge, Pla, Francesc, de Visser, Marianne, Siciliano, Gabriele, Sacconi, Sabrina

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Exon-Skipping in Duchenne Muscular Dystrophy von Takeda, Shin’ichi, Clemens, Paula R., Hoffman, Eric P.

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A Likely Pathogenic variant in the KBTBD13 Gene: A Case Series of Three Patients with Nemaline Myopathy Type 6 von van Kleef, Esmee S B, Bouman, Karlijn, Molenaar, Joery P F, de Winter, Josine M, Duijkers, Floor A M, Eftimov, Filip, Verschuuren-Bemelmans, Corien C, van der Laan, Tineke, Küsters, Benno, Malfatti, Edoardo, Kamsteeg, Erik-Jan, van Engelen, Baziel G M, Ottenheijm, Coen A C, Doorduin, Jonne, Voermans, Nicol C

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