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 24 days ago Drug news

FDA approves Symdeko for use in children with cystic fibrosis who have two copies of the F508del-CFTR mutation or who at least one mutation in the CFTR gene

Vertex Pharmaceuticals Incorporated announced the FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6...

Added 1 month ago Drug news

Vertex has selected the triple combination of VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential regulatory approvals for people ages 12 and older with cystic fibrosis.

Vertex Pharmaceuticals Incorporated announced that it has selected the triple combination of the next-generation corrector VX-445 (elexacaftor), tezacaftor and ivacaftor...

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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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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

End of life care for patients with cystic fibrosis

Palliative care is an approach that improves quality of life for patients and their families facing problems associated with a life-threatening illness. Care planning is particularly important in CF...

Added 8 years ago

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Journal articles

Emerging gene therapies for cystic fibrosis.

Areas covered: We review emerging gene therapies and gene delivery strategies for the treatment of CF particularly viral and non-viral approaches with potential to treat CF.

Added 13 days ago

A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis.

Background: Tezacaftor/ivacaftor is a new treatment option in many regions for patients aged ≥12 years who are homozygous (F/F) or heterozygous for the F508del-CFTR mutation and a residual function (F/RF) mutation.

Added 13 days ago

Delivering on the promise of gene editing for cystic fibrosis.

In this review, we describe a path for translation of gene editing into therapy for cystic fibrosis (CF). Cystic fibrosis results from mutations in the CFTR gene, with one allele predominant in patient populations.

Added 13 days 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 8 days ago

Symdeko in Cystic Fibrosis Patients

This clinical study will enroll 22 participants without the F508del mutation, carrying partial function mutations not approved for Symdeko, and who are not expected to be approved for CFTR modulator treatment in the immediate future.

Added 8 days ago

A Phase 2b Randomised, Placebo Controlled Study of OligoG in Patients With Cystic Fibrosis (CFmised, Double-blind, Parallel-group Study of Alginate Oligosaccharide)

A double-blind, randomised study of OligoG DPI compared to placebo DPI, both on top of standard-of-care, to assess safety, efficacy and tolerability. Adult patients with Cystic Fibrosis will be included in the study.

Added 8 days ago

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Cystic fibrosis: clinical review

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