Cystic fibrosis in the era of precision medicine

Research output: Contribution to journalReview articlepeer-review

20 Scopus citations

Abstract

The treatment of people with cystic fibrosis (CF) has been transformed by the availability of drugs that target the basic chloride defect in the disease. The use of drugs that target specific molecular defects embodies the goals of precision medicine, which incorporate preventive and therapeutic strategies and takes into account differences among individuals. However, the entirety of CF care, from diagnosis to understanding the clinical phenotype and developing a therapeutic strategy, depends on taking into account individual characteristics to achieve optimal outcomes. Future therapies are likely to be even more individualized ushering in a new era of precision medicine.

Original languageEnglish (US)
Pages (from-to)64-72
Number of pages9
JournalPaediatric Respiratory Reviews
Volume25
DOIs
StatePublished - Jan 2018

Keywords

  • CFTR
  • Cystic Fibrosis
  • Genotype
  • Phenotype
  • Precision Medicine

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine

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