Abstract
Relevant, accurate, and sensitive outcome measures are needed to evaluate the effectiveness of new therapeutic interventions or to compare clinical management strategies. In pediatric neuromuscular disorders, these outcome measures must also be well tolerated by children. In clinical research, outcome measures range from biomarkers to measures of clinical function. This chapter systematically reviews outcome measures for use in pediatric neuromuscular research, with a focus on functional measures. We summarize the available data on and experience with each outcome measure, and discuss the potential context for their use. Recent progress in our understanding of the pathobiology of many pediatric neuromuscular disorders has led to several clinical trials evaluating novel interventions. The success of these trials depends on the use of reliable, relevant, and sensitive outcome measures, as does our ability to improve clinical care through evaluating and comparing outcomes.
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