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North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

INTRODUCTION: The aim of this study was to report 36-month longitudinal changes using the North Star Ambulatory Assessment (NSAA) in ambulant patients affected by Duchenne muscular dystrophy amenable to skip exons 44, 45, 51 or 53. MATERIALS AND METHODS: We included 101 patients, 34 had deletions am...

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Опубликовано в: :PLoS One
Главные авторы: Coratti, Giorgia, Pane, Marika, Brogna, Claudia, 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, Hogrel, Jean Yves, Straub, Volker, De Lucia, Silvana, Niks, Erik, Servais, Laurent, De Groot, Imelda, Chesshyre, Mary, Bertini, Enrico, Goemans, Nathalie, Muntoni, Francesco, Mercuri, Eugenio
Формат: Artigo
Язык:Inglês
Опубликовано: Public Library of Science 2021
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC8232423/
https://ncbi.nlm.nih.gov/pubmed/34170974
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0253882
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