Related Experiment Video

Updated: Nov 16, 2025

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

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PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with

Dave P Nichols1, Scott H Donaldson2, Carla A Frederick3

  • 1Seattle Children's Research Institute, Department of Pediatrics, University of Washington, USA.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|February 23, 2021
PubMed

Insights

New cystic fibrosis (CF) modulator therapies, like elexacaftor/tezacaftor/ivacaftor, are transforming care. The PROMISE study investigates their broad impacts on patients aged 6+ in the US.

Area of Science:

  • Medical research
  • Pharmacology
  • Genetics

Background:

  • Highly effective CFTR modulator therapies are now widely available for cystic fibrosis.
  • Observational studies are crucial for understanding the long-term impacts of these treatments.

Purpose of the Study:

  • To investigate the broad impacts of initiating elexacaftor/tezacaftor/ivacaftor therapy.
  • To assess effects on long-term outcomes, patient care needs, and research priorities.
  • To provide insights for a large majority of individuals with cystic fibrosis benefiting from modulator drugs.

Main Methods:

  • The PROMISE study is a large, multi-disciplinary academic research initiative.
  • Focuses on the US population aged 6 years and older starting elexacaftor/tezacaftor/ivacaftor.
  • Organ system-specific investigations are conducted.

Main Results:

  • The study discusses various areas of investigation and their rationales.
  • Highlights remaining questions not addressed by this study alone.
  • Knowledge gained will inform health outcomes and clinical priorities.

Conclusions:

  • This research contributes to understanding the real-world effects of CFTR modulator therapy.
  • Findings will guide future research and clinical care for cystic fibrosis patients.
  • Emphasizes the importance of ongoing studies globally to complement findings.
Keywords:
CftrClinical trialCystic fibrosisDrugElexacaftorIvacaftorModulatorPromiseTezacaftor

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