Revised upper limb module for spinal muscular atrophy: Development of a new module von Mazzone, Elena S., Mayhew, Anna, Montes, Jacqueline, Ramsey, Danielle, Fanelli, Lavinia, Young, Sally Dunaway, Salazar, Rachel, De Sanctis, Roberto, Pasternak, Amy, Glanzman, Allan, Coratti, Giorgia, Civitello, Matthew, Forcina, Nicola, Gee, Richard, Duong, Tina, Pane, Marika, Scoto, Mariacristina, Pera, Maria Carmela, Messina, Sonia, Tennekoon, Gihan, Day, John W., Darras, Basil T., Vivo, Darryl C., Finkel, Richard, Muntoni, Francesco, Mercuri, Eugenio

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Ambulatory function in spinal muscular atrophy: Age-related patterns of progression von Montes, Jacqueline, McDermott, Michael P, Mirek, Elizabeth, Mazzone, Elena S, Main, Marion, Glanzman, Allan M, Duong, Tina, Young, Sally Dunaway, Salazar, Rachel, Pasternak, Amy, Gee, Richard, De Sanctis, Roberto, Coratti, Giorgia, Forcina, Nicola, Fanelli, Lavinia, Ramsey, Danielle, Milev, Evelin, Civitello, Matthew, Pane, Marika, Pera, Maria Carmela, Scoto, Mariacristina, Day, John W, Tennekoon, Gihan, Finkel, Richard S, Darras, Basil T, Muntoni, Francesco, De Vivo, Darryl C, Mercuri, Eugenio

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Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes von Pane, Marika, Mazzone, Elena Stacy, Sivo, Serena, Sormani, Maria Pia, Messina, Sonia, D'Amico, Adele, Carlesi, Adelina, Vita, Gianluca, Fanelli, Lavinia, Berardinelli, Angela, Torrente, Yvan, Lanzillotta, Valentina, Viggiano, Emanuela, D Ambrosio, Paola, Cavallaro, Filippo, Frosini, Silvia, Barp, Andrea, Bonfiglio, Serena, Scalise, Roberta, De Sanctis, Roberto, Rolle, Enrica, Graziano, Alessandra, Magri, Francesca, Palermo, Concetta, Rossi, Francesca, Donati, Maria Alice, Sacchini, Michele, Arnoldi, Maria Teresa, Baranello, Giovanni, Mongini, Tiziana, Pini, Antonella, Battini, Roberta, Pegoraro, Elena, Previtali, Stefano, Bruno, Claudio, Politano, Luisa, Comi, Giacomo P, Bertini, Enrico, Mercuri, Eugenio

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Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data von Pane, Marika, Coratti, Giorgia, Brogna, Claudia, Mazzone, Elena Stacy, Mayhew, Anna, Fanelli, Lavinia, Messina, Sonia, D'Amico, Adele, Catteruccia, Michela, Scutifero, Marianna, Frosini, Silvia, Lanzillotta, Valentina, Colia, Giulia, Cavallaro, Filippo, Rolle, Enrica, De Sanctis, Roberto, Forcina, Nicola, Petillo, Roberta, Barp, Andrea, Gardani, Alice, Pini, Antonella, Monaco, Giulia, D'Angelo, Maria Grazia, Zanin, Riccardo, Vita, Gian Luca, Bruno, Claudio, Mongini, Tiziana, Ricci, Federica, Pegoraro, Elena, Bello, Luca, Berardinelli, Angela, Battini, Roberta, Sansone, Valeria, Albamonte, Emilio, Baranello, Giovanni, Bertini, Enrico, Politano, Luisa, Sormani, Maria Pia, Mercuri, Eugenio

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Functional levels and MRI patterns of muscle involvement in upper limbs in Duchenne muscular dystrophy von Brogna, Claudia, Cristiano, Lara, Tartaglione, Tommaso, Verdolotti, Tommaso, Fanelli, Lavinia, Ficociello, Luana, Tasca, Giorgio, Battini, Roberta, Coratti, Giorgia, Forcina, Nicola, De Santis, Roberto, Norcia, Giulia, Carnicella, Sara, Colosimo, Cesare, Carlier, Pierre, Pane, Marika, Mercuri, Eugenio

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Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy von Mercuri, Eugenio, Coratti, Giorgia, Messina, Sonia, Ricotti, Valeria, Baranello, Giovanni, D'Amico, Adele, Pera, Maria Carmela, Albamonte, Emilio, Sivo, Serena, Mazzone, Elena Stacy, Arnoldi, Maria Teresa, Fanelli, Lavinia, De Sanctis, Roberto, Romeo, Domenico M, Vita, Gian Luca, Battini, Roberta, Bertini, Enrico, Muntoni, Francesco, Pane, Marika

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6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes von Pane, Marika, Mazzone, Elena S, Sormani, Maria Pia, Messina, Sonia, Vita, Gian Luca, Fanelli, Lavinia, Berardinelli, Angela, Torrente, Yvan, D'Amico, Adele, Lanzillotta, Valentina, Viggiano, Emanuela, D'Ambrosio, Paola, Cavallaro, Filippo, Frosini, Silvia, Bello, Luca, Bonfiglio, Serena, Scalise, Roberta, De Sanctis, Roberto, Rolle, Enrica, Bianco, Flaviana, Van der Haawue, Marlene, Magri, Francesca, Palermo, Concetta, Rossi, Francesca, Donati, Maria Alice, Alfonsi, Chiara, Sacchini, Michele, Arnoldi, Maria Teresa, Baranello, Giovanni, Mongini, Tiziana, Pini, Antonella, Battini, Roberta, Pegoraro, Elena, Previtali, Stefano C, Napolitano, Sara, Bruno, Claudio, Politano, Luisa, Comi, Giacomo P, Bertini, Enrico, Morandi, Lucia, Gualandi, Francesca, Ferlini, Alessandra, Goemans, Nathalie, Mercuri, Eugenio

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Reliability of the Performance of Upper Limb assessment in Duchenne muscular dystrophy von Pane, Marika, Mazzone, Elena S, Fanelli, Lavinia, De Sanctis, Roberto, Bianco, Flaviana, Sivo, Serena, D’Amico, Adele, Messina, Sonia, Battini, Roberta, Scutifero, Marianna, Petillo, Roberta, Frosini, Silvia, Scalise, Roberta, Vita, Gianluca, Bruno, Claudio, Pedemonte, Marina, Mongini, Tiziana, Pegoraro, Elena, Brustia, Francesca, Gardani, Alice, Berardinelli, Angela, Lanzillotta, Valentina, Viggiano, Emanuela, Cavallaro, Filippo, Sframeli, Maria, Bello, Luca, Barp, Andrea, Bonfiglio, Serena, Rolle, Enrica, Colia, Giulia, Catteruccia, Michela, Palermo, Concetta, D’Angelo, Grazia, Pini, Antonella, Iotti, Elena, Gorni, Ksenija, Baranello, Giovanni, Morandi, Lucia, Bertini, Enrico, Politano, Luisa, Sormani, MariaPia, Mercuri, Eugenio

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Longitudinal Motor Functional Outcomes and Magnetic Resonance Imaging Patterns of Muscle Involvement in Upper Limbs in Duchenne Muscular Dystrophy von Brogna, Claudia, Cristiano, Lara, Verdolotti, Tommaso, Norcia, Giulia, Ficociello, Luana, Ruiz, Roberta, Coratti, Giorgia, Fanelli, Lavinia, Forcina, Nicola, Petracca, Giorgia, Chieppa, Fabrizia, Tartaglione, Tommaso, Colosimo, Cesare, Pane, Marika, Mercuri, Eugenio

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Timed Rise from Floor as a Predictor of Disease Progression in Duchenne Muscular Dystrophy: An Observational Study von Mazzone, Elena S, Coratti, Giorgia, Sormani, Maria Pia, Messina, Sonia, Pane, Marika, D'Amico, Adele, Colia, Giulia, Fanelli, Lavinia, Berardinelli, Angela, Gardani, Alice, Lanzillotta, Valentina, D'Ambrosio, Paola, Petillo, Roberta, Cavallaro, Filippo, Frosini, Silvia, Bello, Luca, Bonfiglio, Serena, De Sanctis, Roberto, Rolle, Enrica, Forcina, Nicola, Magri, Francesca, Vita, Gianluca, Palermo, Concetta, Donati, Maria Alice, Procopio, Elena, Arnoldi, Maria Teresa, Baranello, Giovanni, Mongini, Tiziana, Pini, Antonella, Battini, Roberta, Pegoraro, Elena, Torrente, Yvan, Previtali, Stefano C, Bruno, Claudio, Politano, Luisa, Comi, Giacomo P, D'Angelo, Maria Grazia, Bertini, Enrico, Mercuri, Eugenio

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Correction: Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes von Pane, Marika, Mazzone, Elena Stacy, Sivo, Serena, Sormani, Maria Pia, Messina, Sonia, D'Amico, Adele, Carlesi, Adelina, Vita, Gianluca, Fanelli, Lavinia, Berardinelli, Angela, Torrente, Yvan, Lanzillotta, Valentina, Viggiano, Emanuela, D'Ambrosio, Paola, Cavallaro, Filippo, Frosini, Silvia, Barp, Andrea, Bonfiglio, Serena, Scalise, Roberta, De Sanctis, Roberto, Rolle, Enrica, Graziano, Alessandra, Magri, Francesca, Palermo, Concetta, Rossi, Francesca, Donati, Maria Alice, Sacchini, Michele, Arnoldi, Maria Teresa, Baranello, Giovanni, Mongini, Tiziana, Pini, Antonella, Battini, Roberta, Pegoraro, Elena, Previtali, Stefano, Bruno, Claudio, Politano, Luisa, Comi, Giacomo P, Bertini, Enrico, Mercuri, Eugenio

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Long-term progression in type II spinal muscular atrophy: A retrospective observational study von Mercuri, Eugenio, Lucibello, Simona, Pera, Maria Carmela, Carnicella, Sara, Coratti, Giorgia, de Sanctis, Roberto, Messina, Sonia, Mazzone, Elena, Forcina, Nicola, Fanelli, Lavinia, Norcia, Giulia, Antonaci, Laura, Frongia, Anna Lia, Pane, Marika

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Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study von Coratti, Giorgia, Bovis, Francesca, Pera, Maria Carmela, Scoto, Mariacristina, Montes, Jacqueline, Pasternak, Amy, Mayhew, Anna, Muni‐Lofra, Robert, Duong, Tina, Rohwer, Annemarie, Dunaway Young, Sally, Civitello, Matthew, Salmin, Francesca, Mizzoni, Irene, Morando, Simone, Pane, Marika, Albamonte, Emilio, D'Amico, Adele, Brolatti, Noemi, Sframeli, Maria, Marini‐Bettolo, Chiara, Sansone, Valeria Ada, Bruno, Claudio, Messina, Sonia, Bertini, Enrico, Baranello, Giovanni, Day, John, Darras, Basil T., De Vivo, Darryl C., Hirano, Michio, Muntoni, Francesco, Finkel, Richard, Mercuri, Eugenio, Antonaci, Laura, De Sanctis, Roberto, Carnicella, Sara, Forcina, Nicola, Norcia, Giulia, Stanca, Giulia, Fanelli, Lavinia, De Luca, Giacomo, Carlesi, Adelina, Colia, Giulia, Bravetti, Chiara, Rossi, Diletta, Rodriguez‐Torres, Rafael

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Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy von Pera, Maria C, Coratti, Giorgia, Forcina, Nicola, Mazzone, Elena S, Scoto, Mariacristina, Montes, Jacqueline, Pasternak, Amy, Mayhew, Anna, Messina, Sonia, Sframeli, Maria, Main, Marion, Lofra, Robert Muni, Duong, Tina, Ramsey, Danielle, Dunaway, Sally, Salazar, Rachel, Fanelli, Lavinia, Civitello, Matthew, de Sanctis, Roberto, Antonaci, Laura, Lapenta, Leonardo, Lucibello, Simona, Pane, Marika, Day, John, Darras, Basil T, De Vivo, Darryl C, Muntoni, Francesco, Finkel, Richard, Mercuri, Eugenio

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Long‐term natural history in type II and III spinal muscular atrophy: a 4‐year international study on the Hammersmith Functional Motor Scale Expanded von Coratti, Giorgia, Bovis, Francesca, Pera, Maria Carmela, Civitello, Matthew, Rohwer, Annemarie, Salmin, Francesca, Glanzman, Allan M., Montes, Jacqueline, Pasternak, Amy, De Sanctis, Roberto, Dunaway Young, Sally, Duong, Tina, Mizzoni, Irene, Milev, Evelin, Sframeli, Maria, Morando, Simone, Albamonte, Emilio, D'Amico, Adele, Catteruccia, Michela, Brolatti, Noemi, Pane, Marika, Scoto, Mariacristina, Messina, Sonia, Escudero, Jesica Exposito, De Waele, Liesbeth, Hirano, Michio, Zolkipli‐Cunningham, Zarazuela, Darras, Basil T., Bertini, Enrico, Nascimiento Osorio, Andres, Bruno, Claudio, Goemans, Natalie, Sansone, Valeria A., Day, John, Baranello, Giovanni, Muntoni, Francesco, Finkel, Richard, Mercuri, Eugenio, Cavallina, Ilaria, Rolle, Enrica, Ricci, Federica, Mongini, Tiziana, Masson, Riccardo, Zanin, Riccardo, Arnoldi, Maria Teresa, Pini, Antonella, Trabacca, Antonio, Rodriguez‐Torres, Rafael S, Forcina, Nicola, Norcia, Giulia, Stanca, Giulia, Carnicella, Sara, Fanelli, Lavinia, Main, Marion, Bravetti, Chiara, Longo, Antonella, Nani, Michela, Antonaci, Laura, Catteruccia, Michela, Materia, Roberto

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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping von Mercuri, E., Seferian, A.M., Servais, L., Deconinck, N., Stevenson, H., Ni, X., Zhang, W., East, L., Yonren, S., Muntoni, F., Deconinck, Nicolas, Van Coster, Rudy, Vanlander, Arnaud, Seferian, Andreea, De Lucia, Silvana, Gidaro, Teresa, Brande, Laura Vanden, Servais, Laurent, Kirschner, Janbernd, Borell, Sabine, Mercuri, Eugenio, Brogna, Claudia, Pane, Marika, Fanelli, Lavinia, Norcia, Giulia, Muntoni, Francesco, Brusa, Chiara, Chesshyre, Mary, Maresh, Kate, Pitchforth, Jaqueline, Schottlaender, Lucia, Scoto, Mariacristina, Silwal, Arpana, Trucco, Fedrica

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Clinical phenotypes and trajectories of disease progression in type 1 spinal muscular atrophy von De Sanctis, Roberto, Pane, Marika, Coratti, Giorgia, Palermo, Concetta, Leone, Daniela, Pera, Maria Carmela, Abiusi, Emanuela, Fiori, Stefania, Forcina, Nicola, Fanelli, Lavinia, Lucibello, Simona, Mazzone, Elena S., Tiziano, Francesco Danilo, Mercuri, Eugenio

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Ultrasound assessment of diaphragmatic function in type 1 spinal muscular atrophy von Buonsenso, Danilo, Berti, Beatrice, Palermo, Concetta, Leone, Daniela, Ferrantini, Gloria, De Sanctis, Roberto, Onesimo, Roberta, Curatola, Antonietta, Fanelli, Lavinia, Forcina, Nicola, Norcia, Giulia, Carnicella, Sara, Lucibello, Simona, Mercuri, Eugenio, Pane, Marika

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Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53 von Brogna, Claudia, Coratti, Giorgia, Pane, Marika, Ricotti, Valeria, Messina, Sonia, D'Amico, Adele, Bruno, Claudio, Vita, Gianluca, Berardinelli, Angela, Mazzone, Elena, Magri, Francesca, Ricci, Federica, Mongini, Tiziana, Battini, Roberta, Bello, Luca, Pegoraro, Elena, Baranello, Giovanni, Previtali, Stefano C, Politano, Luisa, Comi, Giacomo P, Sansone, Valeria A, Donati, Alice, Bertini, Enrico, Muntoni, Francesco, Goemans, Nathalie, Mercuri, Eugenio

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Prognostic factors for tube feeding in type I SMA patients treated with disease-modifying therapies: a cohort study von Pane, Marika, Stanca, Giulia, Coratti, Giorgia, D’ Amico, Adele, Sansone, Valeria Ada, Berti, Beatrice, Fanelli, Lavinia, Albamonte, Emilio, Ausili Cefaro, Carolina, Cerchiari, Antonella, Catteruccia, Michela, De Sanctis, Roberto, Leone, Daniela, Palermo, Concetta, Buchignani, Bianca, Onesimo, Roberta, Kuczynska, Eliza Maria, Tosi, Michele, Pera, Maria Carmela, Bravetti, Chiara, Tiziano, Francesco Danilo, Bertini, Enrico, Mercuri, Eugenio

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