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by McDonald, Craig M and Campbell, Craig and Torricelli, Ricardo Erazo and Finkel, Richard S and Flanigan, Kevin M and Goemans, Nathalie and Heydemann, Peter and Kaminska, Anna and Kirschner, Janbernd and Muntoni, Francesco and Osorio, Andrés Nascimento and Schara, Ulrike and Sejersen, Thomas and Shieh, Perry B and Sweeney, H Lee and Topaloglu, Haluk and Tulinius, Már and Vilchez, Juan J and Voit, Thomas and Wong, Brenda and Elfring, Gary and Kroger, Hans and Luo, Xiaohui and McIntosh, Joseph and Ong, Tuyen and Riebling, Peter and Souza, Marcio and Spiegel, Robert J and Peltz, Stuart W and Mercuri, Eugenio and Alfano, Lindsay N and Eagle, Michelle and James, Meredith K and Lowes, Linda and Mayhew, Anna and Mazzone, Elena S and Nelson, Leslie and Rose, Kristy J and Abdel-Hamid, Hoda Z and Apkon, Susan D and Barohn, Richard J and Bertini, Enrico and Bloetzer, Clemens and de Vaud, Lausanne Canton and Butterfield, Russell J and Chabrol, Brigitte and Chae, Jong-Hee and Jongno-gu, Daehak-ro and Comi, Giacomi Pietro and Darras, Basil T and Dastgir, Jahannaz and Desguerre, Isabelle and Escobar, Raul G and Finanger, Erika and Guglieri, Michela and Hughes, Imelda and Iannaccone, Susan T and Jones, Kristi J and Karachunski, Peter and Kudr, Martin and Lotze, Timothy and Mah, Jean K and Mathews, Katherine and Nevo, Yoram and Parsons, Julie and Péréon, Yann and de Queiroz Campos Araujo, Alexandra Prufer and Renfroe, J Ben and de Resende, Maria Bernadete Dutra and Ryan, Monique and Selby, Kathryn and Tennekoon, Gihan and Vita, Giuseppe and Clinical Evaluator Training Grp and ACT DMD Study Grp and Clinical Evaluator Training Group and ACT DMD Study Group
The Lancet, ISSN 0140-6736, 09/2017, Volume 390, Issue 10101, pp. 1489 - 1498
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 02/2018, Volume 378, Issue 7, pp. 625 - 635
Journal Article
Journal of Clinical Neuromuscular Disease, ISSN 1522-0443, 03/2012, Volume 13, Issue 3, pp. 168 - 189
Journal Article
Journal Article
Annals of Neurology, ISSN 0364-5134, 12/2017, Volume 82, Issue 6, pp. 883 - 891
Journal Article
Neurology, ISSN 0028-3878, 05/2019, Volume 92, Issue 21, pp. e2492 - e2506
OBJECTIVETo report results of intrathecal nusinersen in children with later-onset spinal muscular atrophy (SMA). METHODSAnalyses included children from a phase... 
176 | 228 | 227
Journal Article
Journal of Clinical Neuromuscular Disease, ISSN 1522-0443, 09/2015, Volume 17, Issue 1, pp. 13 - 17
Journal Article
Neuromuscular Disorders, ISSN 0960-8966, 02/2018, Volume 28, Issue 2, pp. 103 - 115
Spinal muscular atrophy (SMA) is a severe neuromuscular disorder due to a defect in the survival motor neuron 1 ( ) gene. Its incidence is approximately 1 in... 
Diagnosis | Nutrition | Orthopedic | Care | Phisotherapy | Spinal muscular atrophy | PHENOTYPIC VARIABILITY | EARLY-ONSET SCOLIOSIS | NEUROSCIENCES | CLINICAL NEUROLOGY | NEUROMUSCULAR SCOLIOSIS | MOTOR-NEURON GENE | GLUCOSE-METABOLISM | CLINICAL-TRIALS | OPERATIVE TREATMENT | CONTROLLED GROWING RODS | CONSENSUS STATEMENT | SMN2 COPY NUMBER | Care and treatment
Journal Article
by Oates, Emily C and Jones, Kristi J and Donkervoort, Sandra and Charlton, Amanda and Brammah, Susan and Smith, John E and Ware, James S and Yau, Kyle S and Swanson, Lindsay C and Whiffin, Nicola and Peduto, Anthony J and Bournazos, Adam and Waddell, Leigh B and Farrar, Michelle A and Sampaio, Hugo A and Teoh, Hooi Ling and Lamont, Phillipa J and Mowat, David and Fitzsimons, Robin B and Corbett, Alastair J and Ryan, Monique M and O'Grady, Gina L and Sandaradura, Sarah A and Ghaoui, Roula and Joshi, Himanshu and Marshall, Jamie L and Nolan, Melinda A and Kaur, Simranpreet and Punetha, Jaya and Töpf, Ana and Harris, Elizabeth and Bakshi, Madhura and Genetti, Casie A and Marttila, Minttu and Werlauff, Ulla and Streichenberger, Nathalie and Pestronk, Alan and Mazanti, Ingrid and Pinner, Jason R and Vuillerot, Carole and Grosmann, Carla and Camacho, Ana and Mohassel, Payam and Leach, Meganne E and Foley, A. Reghan and Bharucha‐Goebel, Diana and Collins, James and Connolly, Anne M and Gilbreath, Heather R and Iannaccone, Susan T and Castro, Diana and Cummings, Beryl B and Webster, Richard I and Lazaro, Leïla and Vissing, John and Coppens, Sandra and Deconinck, Nicolas and Luk, Ho‐Ming and Thomas, Neil H and Foulds, Nicola C and Illingworth, Marjorie A and Ellard, Sian and McLean, Catriona A and Phadke, Rahul and Ravenscroft, Gianina and Witting, Nanna and Hackman, Peter and Richard, Isabelle and Cooper, Sandra T and Kamsteeg, Erik‐Jan and Hoffman, Eric P and Bushby, Kate and Straub, Volker and Udd, Bjarne and Ferreiro, Ana and North, Kathryn N and Clarke, Nigel F and Lek, Monkol and Beggs, Alan H and Bönnemann, Carsten G and MacArthur, Daniel G and Granzier, Henk and Davis, Mark R and Laing, Nigel G
Annals of Neurology, ISSN 0364-5134, 06/2018, Volume 83, Issue 6, pp. 1105 - 1124
Journal Article