The gross motor function measure™ is a valid and sensitive outcome measure for spinal muscular atrophy

Leslie Nelson, Hollis Owens, Linda S. Hynan, Susan T. Iannaccone, Group AmSMART Group

Research output: Contribution to journalArticle

57 Scopus citations

Abstract

Spinal muscular atrophy is a genetic disease of the anterior horn cell with high morbidity rate in childhood. Certain drugs may be of benefit and are in or under consideration for Phase II trials. Outcome measures that are age appropriate and representative of disease activity remain under study. Several have not yet been validated for spinal muscular atrophy. The Gross Motor Function Measure™ is a measure of motor function. We showed previously that the Gross Motor Function Measure™ is a reliable outcome measure to assess motor function in children with spinal muscular atrophy. By collating our data from 40 spinal muscular atrophy patients, ages 5 through 17 years, we now show the validity of the Gross Motor Function Measure™ when compared to Quantitative Muscle Testing and ambulatory status in children with spinal muscular atrophy. The median for Gross Motor Function Measure™ total scores for walkers was 237 (range: 197-261) and for non-walkers, 64 (range: 4-177; P<0.0001) with no distributional overlap. We conclude that the Gross Motor Function Measure™ is valid and sensitive as an outcome measure for clinical trials in pediatric spinal muscular atrophy.

Original languageEnglish (US)
Pages (from-to)374-380
Number of pages7
JournalNeuromuscular Disorders
Volume16
Issue number6
DOIs
StatePublished - Jun 1 2006

Keywords

  • Clinical trials
  • Gross motor function measure
  • Outcome measures
  • Quantitative muscle testing
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

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