Added 3 months ago Drug news

EU approves extension of approval of Kalydeco to infants with CFTR cystic fibrosis.- Vertex Pharma

Vertex Pharmaceuticals announced that the European Commission has granted approval of the label extension for Kalydeco (ivacaftor) to include the...

Added 4 months ago Drug news

Phase III results from two studies of Trikafta for cystic fibrosis published in The New England Journal of Medicine and The Lancet.- Vertex Pharma

Vertex Pharmaceuticals Incorporated announced the concurrent publication in The New England Journal of Medicine (NEJM) and The Lancet of results...

Added 4 months ago Drug news

EMA validates MAA for VX-445 triple combination treatment in cystic fibrosis -Vertex

Vertex Pharmaceuticals has announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX...

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 7 months 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

Advances in gene therapy for cystic fibrosis lung disease.

Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung...

Added 4 months ago

Children With Cystic Fibrosis Are Infected With Multiple Subpopulations of Mycobacterium abscessus With Different Antimicrobial Resistance Profiles.

Background: Children with cystic fibrosis (CF) can develop life-threatening infections of Mycobacterium abscessus. These present a significant clinical challenge, particularly when the strains involved are resistant to antibiotics.

Added 4 months ago

Prevention of chronic infection with Pseudomonas aeruginosa infection in cystic fibrosis.

Purpose of review: This review provides an update on definitions of chronicity of infection, approaches to airway sampling to detect infection, strategies for Pseudomonas aeruginosa eradication, impact of cystic fibrosis...

Added 4 months 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 8 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 5 months 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 5 months ago