title: Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy.
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.
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Eugenio
authors: E
authors: Mercuri
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Giorgia
authors: G
authors: Coratti
authors: Department of Neurosciences and Nemo and Clinical Center, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy.
authors: Sonia
authors: S
authors: Messina
authors: Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, United Kingdom.
authors: Valeria
authors: V
authors: Ricotti
authors: Developmental Neurology Unit, Istituto Neurologico "Besta", Milan, Italy.
authors: Giovanni
authors: G
authors: Baranello
authors: Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Children's Hospital, Rome, Italy.
authors: Adele
authors: A
authors: D'Amico
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Maria Carmela
authors: MC
authors: Pera
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Emilio
authors: E
authors: Albamonte
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Serena
authors: S
authors: Sivo
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Elena Stacy
authors: ES
authors: Mazzone
authors: Developmental Neurology Unit, Istituto Neurologico "Besta", Milan, Italy.
authors: Maria Teresa
authors: MT
authors: Arnoldi
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Lavinia
authors: L
authors: Fanelli
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Roberto
authors: R
authors: De Sanctis
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Domenico M
authors: DM
authors: Romeo
authors: Department of Neurosciences and Nemo and Clinical Center, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy.
authors: Gian Luca
authors: GL
authors: Vita
authors: Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy.
authors: Roberta
authors: R
authors: Battini
authors: Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Children's Hospital, Rome, Italy.
authors: Enrico
authors: E
authors: Bertini
authors: Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, United Kingdom.
authors: Francesco
authors: F
authors: Muntoni
authors: Department of Paediatric Neurology, Catholic University, and Nemo Roma center for neuromuscular disorders, Rome, Italy.
authors: Marika
authors: M
authors: Pane
mesh_terms: D016022:Case-Control Studies; D002657:Child Development / Q000502:physiology; D002675:Child, Preschool; D005080:Exercise Test / Q000379:methods*; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000503:physiopathology*; D011446:Prospective Studies