title: [Upper limb functional assessment scale for children with Duchenne muscular dystrophy and Spinal muscular atrophy].
abstract: Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA) causes significant disability and progressive functional impairment. Readily available instruments that assess functionality, especially in advanced stages of the disease, are required to monitor the progress of the disease and the impact of therapeutic interventions.
  
  
  OBJECTIVE
  To describe the development of a scale to evaluate upper limb function (UL) in patients with DMD and SMA, and describe its validation process, which includes self-training for evaluators.
  
  
  METHODS
  The development of the scale included a review of published scales, an exploratory application of a pilot scale in healthy children and those with DMD, self-training of evaluators in applying the scale using a handbook and video tutorial, and assessment of a group of children with DMD and SMA using the final scale. Reliability was assessed using Cronbach and Kendall concordance and with intra and inter-rater test-retest, and validity with concordance and factorial analysis.
  
  
  RESULTS
  A high level of reliability was observed, with high internal consistency (Cronbach a = 0.97), and inter-rater (Kendall W = 0.96) and intra-rater concordance (r = 0.97 to 0.99). The validity was demonstrated by the absence of significant differences between results by different evaluators with an expert evaluator (F = 0.023, p > .5), and by the factor analysis that showed that four factors account for 85.44% of total variance.
  
  
  CONCLUSIONS
  This scale is a reliable and valid tool for assessing UL functionality in children with DMD and SMA. It is also easily implementable due to the possibility of self-training and the use of simple and inexpensive materials.
authors.affiliation: Unidad de Neurología, División de Pediatría, Escuela de Medicina, Pontificia Universidad Católica de Chile, Santiago, Chile.
authors.forename: Raúl G
authors.initials: RG
authors.lastname: Escobar
authors.affiliation: Laboratorio de Neurorrehabilitación y Enfermedades Neuromusculares Pediátricas, Hospital Clínico, Red de Salud UC-CHRISTUS, Santiago, Chile.
authors.forename: Nayadet
authors.initials: N
authors.lastname: Lucero
authors.affiliation: Escuela de Psicología, Pontificia Universidad Católica de Chile, Santiago, Chile.
authors.forename: Carmen
authors.initials: C
authors.lastname: Solares
authors.affiliation: Escuela de Psicología, Pontificia Universidad Católica de Chile, Santiago, Chile.
authors.forename: Victoria
authors.initials: V
authors.lastname: Espinoza
authors.affiliation: Laboratorio de Neurorrehabilitación y Enfermedades Neuromusculares Pediátricas, Hospital Clínico, Red de Salud UC-CHRISTUS, Santiago, Chile.
authors.forename: Odalie
authors.initials: O
authors.lastname: Moscoso
authors.affiliation: Escuela de Psicología, Pontificia Universidad Católica de Chile, Santiago, Chile.
authors.forename: Polín
authors.initials: P
authors.lastname: Olguín
authors.affiliation: Laboratorio de Neurorrehabilitación y Enfermedades Neuromusculares Pediátricas, Hospital Clínico, Red de Salud UC-CHRISTUS, Santiago, Chile.
authors.forename: Karin T
authors.initials: KT
authors.lastname: Muñoz
authors.affiliation: Escuela de Psicología, Pontificia Universidad Católica de Chile, Santiago, Chile.
authors.forename: Ricardo
authors.initials: R
authors.lastname: Rosas
mesh_terms: D000293:Adolescent; D016022:Case-Control Studies; D002648:Child; D002675:Child, Preschool; D004185:Disability Evaluation; D018450:Disease Progression; D005163:Factor Analysis, Statistical; D005260:Female; D006801:Humans; D008297:Male; D009134:Muscular Atrophy, Spinal / Q000503:physiopathology*; D020388:Muscular Dystrophy, Duchenne / Q000503:physiopathology*; D015588:Observer Variation; D010865:Pilot Projects; D015203:Reproducibility of Results; D012720:Severity of Illness Index; D034941:Upper Extremity