Data from multiple sources - Curated by Marshall Pearce - Last updated 06 June 2017
Cystic fibrosis (CF) is an autosomal recessive, life-limiting, genetically inherited disease. It is a multi-system disorder and results from one of several identified...
Added 10 days ago Drug news
EU approval for Orkambi to treat cystic fibrosis (F508del mutation) in children ages 6 through 11.
Added 1 month ago Drug news
Successful Phase III study of Kalydeco for children aged 1 to 2 years with cystic fibrosis CFTR gene
Added 2 months ago Drug news
Vertex Pharmaceuticals announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase III...
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Specialised CF care has led to a dramatic improvement in survival in CF: in the last four decades, well above...
Added 3 years ago
This review summarizes state-of-the art culture methods and makes recommendations for addition of non-culture based methods in the diagnostic laboratory.
Added 4 years ago
Inert gas washout tests, performed using the single- or multiple-breath washout technique, were first described over 60 years ago. As...
Added 5 years ago
Background: Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show residual function and...
Added 2 months ago
Introduction: Lumacaftor-ivacaftor is indicated for treatment of cystic fibrosis (CF) in patients homozygous for the Phe-508del cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations.
Added 3 months ago
Lumacaftor/ivacaftor, a novel agent for the treatment of cystic fibrosis patients who are homozygous for the F580del CFTR mutation.
Introduction: Cystic Fibrosis (CF) is an autosomal recessive disease affecting up to 90,000 people worldwide. Approximately 73% of patients are homozygous for the F508del cystic fibrosis transmembrane conductance regulator...
Added 3 months ago
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