Doull Iolo
Department of Paediatric Respiratory Medicine and Paediatric, Cystic Fibrosis Centre, Children's Hospital for Wales, Cardiff, CF14 4XN, UK.
Paediatr Respir Rev. 2016 Aug;20 Suppl:18-20. doi: 10.1016/j.prrv.2016.06.008. Epub 2016 Jun 15.
Studies published in the last year have expanded our knowledge of potential disease modifying agents in the treatment of class II, III and IV CFTR mutations, and included the first report of an efficacious gene therapy for CF. There is also an important message on increasing use of conventional chronic therapies even in milder disease, and the pernicious effect of chronic infection on pulmonary function.
去年发表的研究扩展了我们对治疗II、III和IV类CFTR突变潜在疾病修饰剂的认识,其中还包括首篇关于囊性纤维化有效基因疗法的报告。此外,还有一个重要信息,即即使在病情较轻的情况下,传统慢性疗法的使用也在增加,以及慢性感染对肺功能的有害影响。