Traits, trends and hits of orphan drug designations in cystic fibrosis
Author:
Publisher
Elsevier BV
Subject
Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health
Reference45 articles.
1. The future of cystic fibrosis care: a global perspective;Bell;Lancet Respir Med,2020
2. CFTR modulator theratyping: current status, gaps and future directions;Clancy;J Cyst Fibros,2019
3. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation;Accurso;N Engl J Med,2010
4. Lumacaftor–Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR;Wainwright;N Engl J Med,2015
5. Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del;Taylor-Cousar;New Engl J Med,2017
Cited by 2 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. Differential times of submission and approval of CFTR modulators for the treatment of Cystic Fibrosis in the United States and the European Union;Journal of Cystic Fibrosis;2024-08
2. Successes and pitfalls in orphan drug development for sickle cell disease;Blood Advances;2024-05-20
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