NHS England has announced that patients with cystic fibrosis will be able to access triple combination treatment after it struck a deal with Vertex Pharmaceuticals.Gareth IacobucciThe BMJBritish Medical Journal Publishing GroupBMJ
Cystic fibrosis is an autosomal recessive, monogenetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The gene defect was first described 25 years ago and much progress has been made since then in our
The 41-year-old, from Ryton, has spoken out as this week marks 1,000 days since the cystic fibrosis medicine Orkambi was licensed in the UK - but is still unavailable on the NHS. 'The pain isn't as bad but the grief stays for the rest of your life' Woman who lost both siblings ...
Cystic Fibrosis (CF) is an autosomal recessive disease caused by genetic mutations on chromosome 7. The defective gene results in abnormalities in the production and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In h
Cystic Fibrosis research The CF AMR Syndicate aims to address unmet patient needs by linking people with CF with leading experts across industry, academia and the NHS. Together, this cross-sector consortium is identifying and tackling drug discovery hurdles to accelerate the development of CF ...
(2005), Cystic fibrosis and Russell-Silver syndrome in a child with maternal isodisomy of chromosome 7. Pediatr. Pulmonol., 40: 166–168. doi: 10.1002/ppul.20254 Author Information Department of Respiratory Medicine, Great Ormond Street Hospital, London, UK Email: Samatha Sonnappa MRCPCH (son...
P21 Prescribing lvacaftor in paediatric patients with cystic fibrosis (cf) in accordance with the clinical commissioning policy in an nhs trustIntroduction CF is a genetic condition affecting more than 10 800 people in the UK. CF is caused by a mutation in the gene cystic fibrosis trans ...
lung function, risk ofPseudomonas aeruginosacolonisation, and the use of major cystic fibrosis treatment modalities in a UK-wide population cohort, in the context of a the British NHS, a universal healthcare system, free at the point of use (Taylor-Robinson2013; Taylor-Robinson et al.2013a,b...
2Department of Microbiology, Newcastle upon Tyne Hospitals NHS FoundationTrust, Freeman Hospital, Newcastle uponTyne, NE7 7DN, UK. 3Adult Cystic Fibrosis Centre and Northern Oesophago-Gastric Unit Royal Victoria Infirmary, Newcastle upon Tyne, NE1 4LP, UK. Correspondence and requests for materials ...
NHS chief executive Amanda Pritchard described Kaftrio as an "incredible treatment". "Since NHS staff delivered one of the fastest rollouts of Kaftrio in the world just over a year ago, the lives of thousands of patients with cystic fibrosis have been transformed," she said. "Innovative treat...