Data from multiple sources - Curated by Marshall Pearce - Last updated 06 June 2017

Cystic fibrosis

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

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Latest news articles

Added 10 days ago Drug news

First medicine for treatment of underlying cause cystic fibrosis approved.

EU approval for Orkambi to treat cystic fibrosis (F508del mutation) in children ages 6 through 11.

Added 1 month ago Drug news

Cystic Fibrosis study offers potential to modify the course of CF in children

Successful Phase III study of Kalydeco for children aged 1 to 2 years with cystic fibrosis CFTR gene

Added 2 months ago Drug news

Phase III studies of VX 661+ Kalydeco in cystic fibrosis published in NEJM.- Vertex Pharma.

Vertex Pharmaceuticals announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase III...

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Learning Zones

An epgonline.org Learning Zone (LZ) is an area of the site dedicated to providing detailed self-directed medical education about a disease, condition or procedure.

Cystic Fibrosis

Cystic Fibrosis

View disease awareness information, treatment options and European Cystic Fibrosis Society best practice guidelines.

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Hyperammonaemia

Hyperammonaemia

Hyperammonaemia can result in serious neurological damage or death. Could you recognise the signs and symptoms?

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Guidelines

European Cystic Fibrosis Society Standards of Care: Best Practice guidelines

Specialised CF care has led to a dramatic improvement in survival in CF: in the last four decades, well above...

Added 3 years ago

Culture-based diagnostic microbiology in cystic fibrosis: Can we simplify the complexity?

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

Consensus statement for inert gas washout measurement using multiple- and single-breath tests

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

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Journal articles

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.

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

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis.

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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CME

Cystic fibrosis: clinical review

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