Reliability of the Performance of Upper Limb assessment in Duchenne muscular dystrophy.
The Performance of Upper Limb was specifically designed to assess upper limb function in Duchenne muscular dystrophy. The aim of this study was to assess (1) a cohort of typically developing children from the age of 3years onwards in order to identify the age when the activities assessed in the individual items are consistently achieved, and (2) a cohort of 322 Duchenne children and young adults to establish the range of findings at different ages. We collected normative data for the scale validation on 277 typically developing subjects from 3 to 25years old. A full score was consistently achieved by the age of 5years. In the Duchenne cohort there was early involvement of the proximal muscles and a proximal to distal progressive involvement. The scale was capable of measuring small distal movements, related to activities of daily living, even in the oldest and weakest patients. Our data suggest that the assessment can be reliably used in both ambulant and non ambulant Duchenne patients in a multicentric setting and could therefore be considered as an outcome measure for future trials.
Child Neurology and Psychiatry Unit, Rome, Italy.
Marika
M
Pane
Child Neurology and Psychiatry Unit, Rome, Italy.
Elena S
ES
Mazzone
Child Neurology and Psychiatry Unit, Rome, Italy.
Lavinia
L
Fanelli
Child Neurology and Psychiatry Unit, Rome, Italy.
Roberto
R
De Sanctis
Child Neurology and Psychiatry Unit, Rome, Italy.
Flaviana
F
Bianco
Child Neurology and Psychiatry Unit, Rome, Italy.
Serena
S
Sivo
Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Childrens Hospital, Rome, Italy.
Adele
A
D'Amico
Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Italy.
Sonia
S
Messina
Department of Developmental Neuroscience, Stella Maris Institute, Italy.
Roberta
R
Battini
Department of Experimental Medicine, University of Naples, Italy.
Marianna
M
Scutifero
Department of Experimental Medicine, University of Naples, Italy.
Roberta
R
Petillo
Department of Developmental Neuroscience, Stella Maris Institute, Italy.
Silvia
S
Frosini
Child Neurology and Psychiatry Unit, Rome, Italy.
Roberta
R
Scalise
Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Italy.
Gianluca
G
Vita
Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy.
Claudio
C
Bruno
Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy.
Marina
M
Pedemonte
Neuromuscular Center, SG. Battista Hospital, University of Turin, Italy.
Tiziana
T
Mongini
Department of Neurosciences, University of Padua, Italy.
Elena
E
Pegoraro
IRCCS "C. Mondino" Foundation, Pavia, Italy.
Francesca
F
Brustia
IRCCS "C. Mondino" Foundation, Pavia, Italy.
Alice
A
Gardani
IRCCS "C. Mondino" Foundation, Pavia, Italy.
Angela
A
Berardinelli
Neuromuscular Center, SG. Battista Hospital, University of Turin, Italy.
Valentina
V
Lanzillotta
Department of Experimental Medicine, University of Naples, Italy.
Emanuela
E
Viggiano
Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Italy.
Filippo
F
Cavallaro
Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Italy.
Maria
M
Sframeli
IRCCS Eugenio Medea, Bosisio Parini, Italy.
Luca
L
Bello
IRCCS Eugenio Medea, Bosisio Parini, Italy.
Andrea
A
Barp
Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy.
Serena
S
Bonfiglio
Centro Clinico Nemo, Milan, Italy.
Enrica
E
Rolle
Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Childrens Hospital, Rome, Italy.
Giulia
G
Colia
Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Childrens Hospital, Rome, Italy.
Michela
M
Catteruccia
Child Neurology and Psychiatry Unit, Rome, Italy.
Concetta
C
Palermo
IRCCS Eugenio Medea, Bosisio Parini, Italy.
Grazia
G
D'Angelo
Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy.
Antonella
A
Pini
Pediatric Neurology and Neuroradiology Units, Neurological Institute C. Besta, Milan, Italy.
Elena
E
Iotti
Centro Clinico Nemo, Milan, Italy.
Ksenija
K
Gorni
Pediatric Neurology and Neuroradiology Units, Neurological Institute C. Besta, Milan, Italy.
Giovanni
G
Baranello
Pediatric Neurology and Neuroradiology Units, Neurological Institute C. Besta, Milan, Italy.
Lucia
L
Morandi
Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Childrens Hospital, Rome, Italy.
Enrico
E
Bertini
Department of Experimental Medicine, University of Naples, Italy.
Luisa
L
Politano
Biostatistics Unit, Department of Health Sciences, University of Genoa, Italy.
MariaPia
M
Sormani
Child Neurology and Psychiatry Unit, Rome, Italy. Electronic address: mercuri@rm.unicatt.it.
Eugenio
E
Mercuri
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000503:physiopathology*; D063189:Symptom Assessment; D034941:Upper Extremity / Q000503:physiopathology*; D055815:Young Adult
Duchenne muscular dystrophy; Upper limb