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...

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

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

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