Improving complex medical care while awaiting next-generation CFTR potentiators and correctors: The current pipeline of therapeutics

Jennifer L. Goralski, Stephanie Davis

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

While a major target in cystic fibrosis (CF) research in recent years has been the development of corrector and potentiator drugs targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein, these therapies have not yet proven robust enough to replace or eliminate other therapies that have demonstrated improved health outcomes and quality of life in patients with CF. Further, ivacaftor is only indicated for approximately 5% of the US CF population, although the FDA has recently approved lumacaftor/ivacaftor, a combination therapy intended for those homozygous for Phe508del, which should reach a much larger number of patients. This review appraises therapeutics currently available or being studied while we await the next generation of CFTR potentiators and correctors.

Original languageEnglish (US)
Pages (from-to)S66-S73
JournalPediatric Pulmonology
Volume50
DOIs
StatePublished - Oct 1 2015

Fingerprint

Cystic Fibrosis Transmembrane Conductance Regulator
Cystic Fibrosis
Therapeutics
Drug Delivery Systems
Quality of Life
Health
Research
Population
Proteins

Keywords

  • anti-inflammatory
  • antibiotics
  • Cystic fibrosis
  • mucolytics
  • pipeline

ASJC Scopus subject areas

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

Cite this

Improving complex medical care while awaiting next-generation CFTR potentiators and correctors : The current pipeline of therapeutics. / Goralski, Jennifer L.; Davis, Stephanie.

In: Pediatric Pulmonology, Vol. 50, 01.10.2015, p. S66-S73.

Research output: Contribution to journalArticle

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