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...
Added 5 hours ago Drug news
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...
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.
View disease awareness information, treatment options and European Cystic Fibrosis Society best practice guidelines.
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.
'New IBD assessment tools at a glance' - an educational symposium sponsored by Sandoz.
Explore the pathophysiology, epidemiology and multi-system symptoms associated with hereditary ATTR amyloidosis, as well as how to achieve an early and accurate diagnosis.
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
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
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
CFTR has mainly been studied in epithelial cells although it is also functional and expressed in other cell types including endothelial cells. The present review summarizes current knowledge on the role of the endothelium in CF.
Added 1 day ago
Physical activity and associations with clinical outcome measures in adults with cystic fibrosis; a systematic review.
Background: Physical activity (PA) is important in the management of Cystic Fibrosis (CF) and is associated with a number of beneficial effects. PA assessment is not commonplace or consistent in clinical practice...
Added 2 days ago
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 2 months ago
A Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With CF (ANAKIN)
- 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 2 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 2 days ago
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