Year in review 2019: Neuromuscular diseases.
PEDIATRIC PULMONOLOGY(2020)
摘要
Neuromuscular cardiopulmonary medicine is entering a new and exciting phase, with studies that assess the respiratory effect of emerging genetic and molecular therapies. In this year's neuromuscular Year in Review, we focus on Duchenne muscular dystrophy (DMD), reviewing studies that evaluate the respiratory effect of eteplirsen, the cardiopulmonary effects of ataluren, and a study comparing the use of spironolactone with eplerenone for the treatment of DMD-related cardiomyopathy.
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关键词
clinical trials,neuromuscular disorders,pharmacology,pulmonary function testing
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