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

doi:10.1371/journal.pone.0253882

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Article

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

PloS one - 1 Jan 2021

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

Abstract excerpt

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 amenable to skip exon 44, 25 exon 45, 19 exon 51, and 28 exon 53, not recruited in any ongoing clinical trials. Five...

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PMID: 34170974
DOI: 10.1371/journal.pone.0253882
PMCID: PMC8232423

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