Added 26 days 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...

Added 1 month 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 2 months ago Congress news

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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

Channelopathies.

Channelopathies are a heterogeneous group of disorders resulting from the dysfunction of ion channels located in the membranes of all cells and many cellular organelles. These include diseases of the nervous system (e.g., generalized epilepsy...

Added 3 days ago

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 1 month 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 1 month ago

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 1 month 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 1 month 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 1 month ago