VOCAL: An observational study of ivacaftor for people with cystic fibrosis and selected non–G551D-CFTR gating mutations

Author:

Simmonds Nicholas J.ORCID,van der Ent C. Kors,Colombo Carla,Kinnman NilsORCID,DeSouza Cynthia,Thorat TejaORCID,Chew Marci L.,Chandarana Keval,Castellani CarloORCID

Publisher

Elsevier BV

Subject

Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health

Reference30 articles.

1. European Cystic Fibrosis Society patient registry annual data report 2018. [Internet];Zolin,2020

2. Progress in therapies for cystic fibrosis;De Boeck;Lancet Respir Med,2016

3. Ivacaftor potentiation of multiple CFTR channels with gating mutations;Yu;J Cyst Fibros,2012

4. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation;Ramsey;N Engl J Med,2011

5. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation;Davies;Am J Respir Crit Care Med,2013

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1. Marginal versus conditional rate estimation for count and recurrent event data with an estimand framework;Contemporary Clinical Trials;2024-03

2. Ivacaftor;Reactions Weekly;2023-04-08

3. Progress and challenges in fungal lung disease in cystic fibrosis;Current Opinion in Pulmonary Medicine;2022-09-19

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