Clinical outcome assessments in Duchenne muscular dystrophy and spinal muscular atrophy: past, present and future

Leslie L. Nelson, Susan T. Iannaccone

Research output: Contribution to journalReview articlepeer-review

Abstract

Scores and scales used in pediatric motor development for neuromuscular disorders have evolved greatly since the beginning of their development. In this review we provide a brief history of scales used in pediatric patients with neuromuscular disorders and an update regarding the advancement of the scales commonly used in patients with spinal muscular atrophy and Duchenne muscular dystrophy. We focus on the collaborative effort that has led to the development of outcomes and speak to the possible future of Clinical Outcome Assessments.

Original languageEnglish (US)
Pages (from-to)1028-1037
Number of pages10
JournalNeuromuscular Disorders
Volume31
Issue number10
DOIs
StatePublished - Oct 2021

Keywords

  • clinical outcome assessment
  • Duchenne muscular dystrophy
  • functional outcome
  • motor development
  • motor performance
  • spinal muscular atrophy

ASJC Scopus subject areas

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

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