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目前对接受CFTR调节剂治疗的囊性纤维化患者进行长期评估、监测和管理的策略。

Current strategies for the long-term assessment, monitoring, and management of cystic fibrosis patients treated with CFTR modulator therapy.

作者信息

Elborn J Stuart, Davies Jane, Mall Marcus A, Flume Patrick A, Plant Barry

机构信息

Queen's University of Belfast, Belfast, Northern Ireland, United Kingdom.

Imperial College London, Royal Brompton and Harefield NHS Foundation Trust, London, United Kingdom.

出版信息

J Cyst Fibros. 2017 Jan;16(1):163-164. doi: 10.1016/j.jcf.2016.11.009. Epub 2016 Dec 13.

Abstract

The content for this activity is based on the satellite symposium, "Current Strategies for the Long-term Assessment, Monitoring, and Management for Cystic Fibrosis Patients Treated with CFTR Modulator Therapy" that was presented at the 39th European Cystic Fibrosis Society Conference on June 10, 2016 (Online access: http://courses.elseviercme.com/ecfs2016e/619e). The emergence of novel targeted agents, that directly correct CFTR loss function alleles, has created new treatment opportunities for patients with cystic fibrosis with advanced disease. Knowledge of the role of these agents in the clinical setting is quickly evolving and will require physicians to stay acquainted with the latest data as well as evidence-based treatment guidelines in order to achieve optimized cystic fibrosis patient care. Ideally, after diagnosis, a personalized approach would be adapted and tailored to the patient through genome-informed medicine. However, due to the relative recentness of genomic-based therapeutics, physicians may have a limited knowledge base regarding these new treatment options and how to best incorporate these agents into patient management plans. Although cystic fibrosis is still largely regarded as a pediatric disease, the median survival for patients is 35years of age. Consequently, pediatric-to-adult cystic fibrosis care programs would allow suitable preparation time for this transition and develop a standardized group of self-care and management skills.

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