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Elucidating progression of early cystic fibrosis lung disease
European Respiratory Journal ( IF 16.6 ) Pub Date : 2017-11-01 , DOI: 10.1183/13993003.01916-2017
Kathryn Ramsey , Felix Ratjen , Philipp Latzin

Widespread implementation of newborn screening has allowed early diagnosis and disease detection in infants with cystic fibrosis (CF). Early surveillance has shown that pulmonary infection and inflammation occur early in life, and are associated with the development of structural disease as well as lung function decline in childhood [1, 2]. With the advent of disease-modifying therapies for CF, and evidence of their efficacy in adults and adolescents [3], there is a need for sensitive outcome measures to monitor efficacy and safety of these therapies in infancy. Findings that infants with CF followed up regularly have mild, transient lung function deficits are encouraging http://ow.ly/SOKN30fRn3I

中文翻译:

阐明早期囊性纤维化肺病的进展

新生儿筛查的广泛实施使得囊性纤维化 (CF) 婴儿的早期诊断和疾病检测成为可能。早期监测表明,肺部感染和炎症发生在生命早期,并与儿童期结构性疾病的发展以及肺功能下降有关 [1, 2]。随着 CF 疾病改善疗法的出现,以及它们在成人和青少年中的疗效证据[3],需要敏感的结局指标来监测这些疗法在婴儿期的疗效和安全性。定期随访 CF 婴儿有轻度、暂时​​性肺功能缺陷的发现令人鼓舞 http://ow.ly/SOKN30fRn3I
更新日期:2017-11-01
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