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 2 days ago Drug news

CHMP recommends approval of Kalydeco in cystic fibrosis CFTR gene mutation.- Vertex Pharma

Vertex Pharmaceuticals (Europe) Limited announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted...

Added 1 month ago Drug news

FDA accepts elexacaftor, tezacaftor and ivacaftor, triple therapy, to treat cystic fibrosis and assigned a PDUFA date of 19 March 2020

Vertex Pharmaceuticals Incorporated announced the FDA accepted its New Drug Application (NDA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple...

Added 2 months ago Drug news

Vertex Pharma files NDA at FDA for elexacaftor, tezacaftor and ivacaftor combination for F508del mutation cystic fibrosis.

Vertex Pharmaceuticals Incorporated announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor...

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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.

Inflammatory Bowel Disease Knowledge Centre

Inflammatory Bowel Disease Knowledge Centre

The Inflammatory Bowel Disease (IBD) Knowledge Centre contains key information relating to the epidemiology and pathophysiology of Crohn’s disease and ulcerative colitis, highlighting prevalence, impact and unmet needs and the underlying inflammatory processes that drive IBD, considering some of the key inflammatory pathways.

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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 1 month 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

Prevention of drug-related complications in cystic fibrosis.

Purpose of review: Due to continuous development of new drugs and better treatment strategies, survival of patients with cystic fibrosis has changed dramatically. Recently, targeted therapy of cystic fibrosis transmembrane...

Added 2 hours ago

Current Practices and Potential Nanotechnology Perspectives for Pain Related to Cystic Fibrosis.

Pain is a complex, multidimensional process that negatively affects physical and mental functioning, clinical outcomes, quality of life, and productivity for cystic fibrosis (CF) patients. CF is an inherited multi-system disease that requires...

Added 2 hours ago

The future of cystic fibrosis care: a global perspective.

The past six decades have seen remarkable improvements in health outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young children. However, although life expectancy for people with cystic fibrosis...

Added 2 hours 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 3 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 4 days 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 4 days ago

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Cystic fibrosis: clinical review

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MIMS Learning - Monthly Index of Medical Specialities
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