Related Experiment Video

Updated: Mar 1, 2026

Implementation of Non-invasive Point of Care Transient Elastography for Evaluation of Liver Disease in Pediatric Populations with Cystic Fibrosis
05:56

Implementation of Non-invasive Point of Care Transient Elastography for Evaluation of Liver Disease in Pediatric Populations with Cystic Fibrosis

Published on: August 29, 2025

608

Disease-modifying drug therapy in cystic fibrosis

Katharine Harman1, Rebecca Dobra1, Jane C Davies1

  • 1Imperial College London & Royal Brompton & Harefield NHS Trust, London, UK.

Paediatric Respiratory Reviews
|June 7, 2017
PubMed

Insights

Small molecule drugs targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein offer a new disease-modifying approach for cystic fibrosis treatment. This review covers progress and challenges in CFTR modulator therapies.

Area of Science:

  • Biochemistry
  • Pharmacology
  • Genetics

Background:

  • Cystic Fibrosis (CF) remains a significant health burden despite treatment advances.
  • Early mortality and symptom severity persist in CF patients.
  • The CFTR protein is a key target for novel therapeutic strategies.

Purpose of the Study:

  • To review the progress in small molecule drug development for CF.
  • To highlight current and future challenges in CFTR modulator therapies.
  • To discuss the potential of disease-modifying treatments for CF.

Main Methods:

  • Literature review of recent advancements in CFTR modulator research.
  • Analysis of the impact of small molecule drugs on CF treatment.
  • Discussion of therapeutic challenges and future directions.

Main Results:

  • A new era of CFTR modulator therapies has emerged in the last decade.
  • Small molecule drugs offer a potential disease-modifying approach to CF treatment.
  • Significant progress has been made, but challenges remain.

Conclusions:

  • CFTR modulator therapies represent a paradigm shift in cystic fibrosis management.
  • Continued research is crucial to overcome existing challenges and optimize treatment.
  • These therapies hold promise for improving patient outcomes and reducing disease burden.
Keywords:
CFTRcorrectorpotentiatorread-through

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