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

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. We included 101 patients, 34 had deletions amenable to skip exon 44, 25 exon 45, 1...

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Veröffentlicht in:	PloS one 2021-06, Vol.16 (6), p.e0253882-e0253882
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Ambulatory assessment Analysis Biology and Life Sciences Biomedical research Boys Child Children & youth Childrens health Clinical trials Consortia Diagnosis Disease Progression Duchenne muscular dystrophy Duchenne's muscular dystrophy Dystrophin/genetics Dystrophy Exon (Molecular genetics) Exons Exons/genetics Follow-Up Studies Genetic aspects Health risks Hospitals Human health sciences Humans Longitudinal Studies Male Medicine and Health Sciences Men Muscular dystrophy Muscular Dystrophy, Duchenne/genetics/pathology Mutation Mutation/genetics Neurologie Neurology Neurosciences Pathophysiology Pediatrics Physical Sciences Public health Pédiatrie Sciences de la santé humaine Severity of Illness Index Subgroups Walking/physiology
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