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 1 month ago Drug news

EU approves extension of approval of Kalydeco to infants with CFTR cystic fibrosis.- Vertex Pharma

Vertex Pharmaceuticals announced that the European Commission has granted approval of the label extension for Kalydeco (ivacaftor) to include the...

Added 2 months ago Drug news

Phase III results from two studies of Trikafta for cystic fibrosis published in The New England Journal of Medicine and The Lancet.- Vertex Pharma

Vertex Pharmaceuticals Incorporated announced the concurrent publication in The New England Journal of Medicine (NEJM) and The Lancet of results...

Added 2 months ago Drug news

EMA validates MAA for VX-445 triple combination treatment in cystic fibrosis -Vertex

Vertex Pharmaceuticals has announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX...

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Learning Zones

An 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 Knowledge Centre

Cystic Fibrosis Knowledge Centre

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

Anti-integrins in IBD

Anti-integrins in IBD

See presentations from a satellite symposium presented at UEG Week 2019.

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End of life care for infants, children and young people with life-limiting conditions: planning and management

This guideline covers the planning and management of end of life and palliative care in for infants, children and young people (aged 0–17 years) with life-limiting conditions. It aims to involve children, young people and their families...

Added 4 months ago

Cystic fibrosis: diagnosis and management

This guideline covers diagnosing and managing cystic fibrosis. It specifies how to monitor the condition and manage the symptoms to improve quality of life.

Added 1 year ago

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 what was seen in the general population over the same period.

Added 5 years ago

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Journal articles

Advances in gene therapy for cystic fibrosis lung disease.

Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung...

Added 1 month ago

Children With Cystic Fibrosis Are Infected With Multiple Subpopulations of Mycobacterium abscessus With Different Antimicrobial Resistance Profiles.

Background: Children with cystic fibrosis (CF) can develop life-threatening infections of Mycobacterium abscessus. These present a significant clinical challenge, particularly when the strains involved are resistant to antibiotics.

Added 1 month ago

Prevention of chronic infection with Pseudomonas aeruginosa infection in cystic fibrosis.

Purpose of review: This review provides an update on definitions of chronicity of infection, approaches to airway sampling to detect infection, strategies for Pseudomonas aeruginosa eradication, impact of cystic fibrosis...

Added 1 month ago

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Clinical trials

A Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With CF (ANAKIN)

Primary Ojective:
- To evaluate efficacy of treatment with anakinra in subjects with CF who are ≥ 12 years of age by means of lung clearance index (LCI).

Added 6 months ago

A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for F508del.

Added 3 months ago

A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del...

Added 3 months ago

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Cystic fibrosis: clinical review

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MIMS Learning - Monthly Index of Medical Specialities
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