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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Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy von Trundell, Dylan, Le Scouiller, Stephanie, Le Goff, Laure, Gorni, Ksenija, Vuillerot, Carole

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Responsiveness of the Motor Function Measure in Patients With Spinal Muscular Atrophy von Vuillerot, Carole, MD, PhD, Payan, Christine, MD, Iwaz, Jean, PhD, Ecochard, René, MD, PhD, Bérard, Carole, MD

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Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial von Bertini, Enrico, Prof, Dessaud, Eric, PhD, Mercuri, Eugenio, Prof, Muntoni, Francesco, Prof, Kirschner, Janbernd, Prof, Reid, Carol, PhD, Lusakowska, Anna, MD, Comi, Giacomo P, Prof, Cuisset, Jean-Marie, MD, Abitbol, Jean-Louis, MD, Scherrer, Bruno, PhD, Ducray, Patricia Sanwald, PhD, Buchbjerg, Jeppe, MSc, Vianna, Eduardo, PhD, van der Pol, W Ludo, MD, Vuillerot, Carole, MD, Blaettler, Thomas, MD, Fontoura, Paulo, MD

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Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study von Audic, Frédérique, de la Banda, Marta Gomez Garcia, Bernoux, Delphine, Ramirez-Garcia, Paola, Durigneux, Julien, Barnerias, Christine, Isapof, Arnaud, Cuisset, Jean-Marie, Cances, Claude, Richelme, Christian, Vuillerot, Carole, Laugel, Vincent, Ropars, Juliette, Altuzarra, Cécilia, Espil-Taris, Caroline, Walther-Louvier, Ulrike, Sabouraud, Pascal, Chouchane, Mondher, Vanhulle, Catherine, Trommsdorff, Valérie, Pervillé, Anne, Testard, Hervé, Lagrue, Emmanuelle, Sarret, Catherine, Avice, Anne-Laude, Beze-Beyrie, Pierre, Pauly, Vanessa, Quijano-Roy, Susana, Chabrol, Brigitte, Desguerre, Isabelle

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Motor Function Measure: Validation of a Short Form for Young Children With Neuromuscular Diseases von de Lattre, Capucine, MD, Payan, Christine, MD, Vuillerot, Carole, MD, PhD, Rippert, Pascal, PhD, de Castro, Denis, MD, Bérard, Carole, MD, Poirot, Isabelle, MD, PhD

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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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Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint von Servais, Laurent, Strijbos, Paul, Poleur, Margaux, Mirea, Andrada, Butoianu, Nina, Sansone, Valeria A., Vuillerot, Carole, Schara-Schmidt, Ulrike, Scoto, Mariacristina, Seferian, Andreea M., Previtali, Stefano C., Tulinius, Már, Nascimento, Andrés, Furlong, Pat, Singh, Teji, Dreghici, Roxana Donisa, Goemans, Nathalie, Mercuri, Eugenio, Straub, Volker, Ormazabal, Maitea Guridi, Braid, Jessica, Muntoni, Francesco, Tricot, Alexis, Annoussamy, Mélanie, Eggenspieler, Damien

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Muscle Activation during Gait in Children with Duchenne Muscular Dystrophy von Ropars, Juliette, Lempereur, Mathieu, Vuillerot, Carole, Tiffreau, Vincent, Peudenier, Sylviane, Cuisset, Jean-Marie, Pereon, Yann, Leboeuf, Fabien, Delporte, Ludovic, Delpierre, Yannick, Gross, Raphaël, Brochard, Sylvain

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Validity and Reliability of the 32-Item Motor Function Measure in 2- to 5-Year-Olds with Neuromuscular Disorders and 2- to 25-Year-Olds with Spinal Muscular Atrophy von Trundell, Dylan, Le Scouiller, Stephanie, Gorni, Ksenija, Seabrook, Timothy, Vuillerot, Carole

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Spinal muscular atrophy is also a disorder of spermatogenesis von Magot, Armelle, Reignier, Arnaud, Binois, Olivier, Bedat-Millet, Anne Laure, Davion, Jean-Baptiste, Debergé, Louise, Ghorab, Karima, Guyant, Lucie, Laheranne, Émilie, Laforet, Pascal, Lefeuvre, Claire, Mallaret, Martial, Michaud, Maud, Omar, Chahla, Nadaj-Pakleza, Aleksandra, Nicolas, Guillaume, Noury, Jean Baptiste, Pegat, Antoine, Péré, Morgane, Salort-Campana, Emmanuelle, Sole, Guilhem, Spinazzi, Marco, Tard, Céline, Vuillerot, Carole, Péréon, Yann

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A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data von Duong, Tina, Staunton, Hannah, Braid, Jessica, Barriere, Aurelie, Trzaskoma, Ben, Gao, Ling, Willgoss, Tom, Cruz, Rosangel, Gusset, Nicole, Gorni, Ksenija, Randhawa, Sharan, Yang, Lida, Vuillerot, Carole

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Confirmatory validation of the french version of the Duchenne Muscular Dystrophy module of the pediatric quality of life inventory (PedsQLTM3.0DMDfv) von Wallach, Elisabeth, Ehlinger, Virginie, Biotteau, Maelle, Walther-Louvier, Ulrike, Péréon, Yann, Vuillerot, Carole, Fontaine, Stephanie, Sabouraud, Pascal, Espil-Taris, Caroline, Cuisset, Jean-Marie, Laugel, Vincent, Baudou, Eloïse, Arnaud, Catherine, Cances, Claude

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Multidisciplinary team meetings in treatment of spinal muscular atrophy adult patients: a real-life observatory for innovative treatments von Salort-Campana, Emmanuelle, Solé, Guilhem, Magot, Armelle, Tard, Céline, Noury, Jean-Baptiste, Behin, Anthony, De La Cruz, Elisa, Boyer, François, Lefeuvre, Claire, Masingue, Marion, Debergé, Louise, Finet, Armelle, Brison, Mélanie, Spinazzi, Marco, Pegat, Antoine, Sacconi, Sabrina, Malfatti, Edoardo, Choumert, Ariane, Bellance, Rémi, Bedat-Millet, Anne-Laure, Feasson, Léonard, Vuillerot, Carole, Jacquin-Piques, Agnès, Michaud, Maud, Pereon, Yann, Stojkovic, Tanya, Laforêt, Pascal, Attarian, Shahram, Cintas, Pascal

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Responsiveness of the Motor Function Measure in Neuromuscular Diseases von Vuillerot, Carole, MD, PhD, Payan, Christine, MD, Girardot, Françoise, SRP, Fermanian, Jacques, MD, PhD, Iwaz, Jean, PhD, Bérard, Carole, MD, Ecochard, René, MD, PhD

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Use of MFM-20 to monitor SMA types 1 and 2 patients treated with nusinersen von Le Goff, Laure, Seferian, Andreea, Phelep, Aurelie, Rippert, Pascal, Mathieu, Marie-Laure, Cances, Claude, de Lattre, Capucine, Durigneux, Julien, Gousse, Gaelle, Vincent-Genod, Dominique, Ribault, Shams, Gomez Garcia de la Banda, Marta, Quijano-Roy, Susana, Sarret, Catherine, Servais, Laurent, Vuillerot, Carole

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Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a... von Duong, Tina, Braid, Jessica, Staunton, Hannah, Barriere, Aurelie, Petridis, Fani, Reithinger, Johannes, Cruz, Rosangel, Jarecki, Jill, De Lemus, Mencia, Gusset, Nicole, Broekgaarden, Ria, Randhawa, Sharan, Flynn, Jessica, Arbuckle, Rob, Reif, Sonia, Yang, Lida, De Martini, Angela, Vuillerot, Carole

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Use of assistive technology to assess distal motor function in subjects with neuromuscular disease von Vincent-Genod, Dominique, Roche, Sylvain, Barrière, Aurélie, de Lattre, Capucine, Tinat, Marie, Venema, Eelke, Lagrange, Emmeline, Gomes Lisboa de Souza, Adriana, Thomann, Guillaume, Coton, Justine, Gautheron, Vincent, Féasson, Léonard, Rippert, Pascal, Vuillerot, Carole

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Correction to: Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers’... von Duong, Tina, Braid, Jessica, Staunton, Hannah, Barriere, Aurelie, Petridis, Fani, Reithinger, Johannes, Cruz, Rosangel, Jarecki, Jill, De Lemus, Mencia, Gusset, Nicole, Broekgaarden, Ria, Randhawa, Sharan, Flynn, Jessica, Arbuckle, Rob, Reif, Sonia, Yang, Lida, De Martini, Angela, Vuillerot, Carole

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Motor and respiratory heterogeneity in Duchenne patients: Implication for clinical trials von Humbertclaude, Véronique, Hamroun, Dalil, Bezzou, Kamel, Bérard, Carole, Boespflug-Tanguy, Odile, Bommelaer, Christine, Campana-Salort, Emmanuelle, Cances, Claude, Chabrol, Brigitte, Commare, Marie-Christine, Cuisset, Jean-Marie, de Lattre, Capucine, Desnuelle, Claude, Echenne, Bernard, Halbert, Cécile, Jonquet, Olivier, Labarre-Vila, Annick, N’Guyen-Morel, Marie-Ange, Pages, Michel, Pepin, Jean-Louis, Petitjean, Thierry, Pouget, Jean, Ollagnon-Roman, Elisabeth, Richelme, Christian, Rivier, François, Sacconi, Sabrina, Tiffreau, Vincent, Vuillerot, Carole, Picot, Marie-Christine, Claustres, Mireille, Béroud, Christophe, Tuffery-Giraud, Sylvie

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