Advances in the restoration of CFTR in children and pwCF with “mild disease”

Malena Cohen-Cymberknoh

doi:10.1002/ppul.27359

Advances in the restoration of CFTR in children and pwCF with “mild disease”

Malena Cohen-Cymberknoh^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

Abstract

Cystic fibrosis (CF) is a progressive genetic disorder, with lung disease being the main cause of morbidity and mortality. While advances in treatment have extended life expectancy, lung function still declines over time. Early inflammation and chronic infection, particularly with pseudomonas, worsen outcomes. Current management focuses on nutrition, airway clearance and infection control, but CFTR modulators directly target the genetic defect, improving lung function and reducing pulmonary exacerbations. Early use of CFTR modulators can alter the course of the disease.

Original language	English
Pages (from-to)	S84-S85
Journal	Pediatric Pulmonology
Volume	60
Issue number	S1
DOIs	https://doi.org/10.1002/ppul.27359
State	Published - Mar 2025
Externally published	Yes

Bibliographical note

Publisher Copyright:
© 2024 The Author(s). Pediatric Pulmonology published by Wiley Periodicals LLC.

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Keywords

CFTR modulators
airway Inflammation
cystic fibrosis
lung disease progression
pulmonary exacerbations

Access to Document

10.1002/ppul.27359

Cite this

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abstract = "Cystic fibrosis (CF) is a progressive genetic disorder, with lung disease being the main cause of morbidity and mortality. While advances in treatment have extended life expectancy, lung function still declines over time. Early inflammation and chronic infection, particularly with pseudomonas, worsen outcomes. Current management focuses on nutrition, airway clearance and infection control, but CFTR modulators directly target the genetic defect, improving lung function and reducing pulmonary exacerbations. Early use of CFTR modulators can alter the course of the disease.",

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Advances in the restoration of CFTR in children and pwCF with “mild disease”

Abstract

Bibliographical note

UN SDGs

Keywords

Access to Document

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Cite this