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

Leslie L. Nelson; Susan T. Iannaccone

doi:10.1016/j.nmd.2021.07.015

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 journal › Review article › peer-review

2 Scopus citations

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 language	English (US)
Pages (from-to)	1028-1037
Number of pages	10
Journal	Neuromuscular Disorders
Volume	31
Issue number	10
DOIs	https://doi.org/10.1016/j.nmd.2021.07.015
State	Published - Oct 2021

Keywords

Duchenne muscular dystrophy
clinical outcome assessment
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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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.",

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AB - 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.

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KW - clinical outcome assessment

KW - functional outcome

KW - motor development

KW - motor performance

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

Abstract

Keywords

ASJC Scopus subject areas

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