Lumacaftor/ivacaftor reduces exacerbations in adults homozygous for Phe508del mutation with severe lung disease
Author:
Funder
Vertex Pharmaceuticals
Publisher
Elsevier BV
Subject
Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health
Reference14 articles.
1. Cystic fibrosis transmembrane conductance regulator dysfunction and its treatment;Hull;J R Soc Med,2012
2. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809;Van Goor;Proc Natl Acad Sci USA,2011
3. Lumacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR;Wainwright;N Engl J Med,2015
4. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the gly551asp-cftr mutation: a phase 3, open-label extension study (PERSIST);McKone;Lancet Respir Med,2014
5. Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis;Elborn;Lancet Respir Med,2016
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1. Impact of lumacaftor/ivacaftor on nutrition and growth in modulator-naïve children over 24 weeks;Journal of Cystic Fibrosis;2024-07
2. Effectiveness of lumacaftor/ivacaftor initiation in children with cystic fibrosis aged 2 through 5 years on disease progression: Interim results from an ongoing registry-based study;Journal of Cystic Fibrosis;2024-05
3. Use of CFTR modulators in special populations, part 2: Severe lung disease;Pediatric Pulmonology;2023-09-20
4. CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis;Cells;2022-04-06
5. Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study;Journal of Cystic Fibrosis;2022-01
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