Revised north star ambulatory assessment for young boys with Duchenne muscular dystrophy

Eugenio Maria Mercuri, Domenico Marco Romeo, Marika Pane, Giorgia Coratti, Giovanni Baranello, Adele D'Amico, Maria Carmela Pera, Emilio Albamonte, Serena Sivo, Elena Stacy Mazzone, Roberto De Sanctis, Roberta Battini, Enrico Silvio Bertini, Sonia Messina, Valeria Ricotti, Maria Teresa Arnoldi, Gian Luca Vita, Enrico Bertini, Francesco Muntoni

Risultato della ricerca: Contributo in rivistaArticolo in rivista

17 Citazioni (Scopus)

Abstract

The advent of therapeutic approaches for Duchenne muscular dystrophy (DMD) has highlighted the need to identify reliable outcome measures for young boys with DMD. The aim of this study was to develop a revised version of the North Star Ambulatory Assessment (NSAA) suitable for boys between the age of 3 and 5 years by identifying age appropriate items and revising the scoring system accordingly. Using the scale in 171 controls between the age of 2.9 and 4.8 years, we identified items that were appropriate at different age points. An item was defined as age appropriate if it was completed, achieving a full score, by at least 85% of the typically developing boys at that age. At 3 years (±3months) there were only 8 items that were age appropriate, at 3 years and 6 months there were 13 items while by the age of 4 years all 17 items were appropriate. A revised version of the scale was developed with items ordered according to the age when they could be reliably performed. The application of the revised version of the scale to data collected in young DMD boys showed that very few of the DMD boys were able to complete with a full score all the age appropriate items. In conclusion, our study suggests that a revised version of the NSAA can be used in boys from the age of 3 years to obtain information on how young DMD boys acquire new abilities and how this correlates with their peers.
Lingua originaleEnglish
pagine (da-a)e0160195-e0160195
RivistaPLoS One
Volume11
DOI
Stato di pubblicazionePubblicato - 2016

Keywords

  • Agricultural and Biological Sciences (all)
  • Biochemistry, Genetics and Molecular Biology (all)
  • Medicine (all)

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