Tracking early cystic fibrosis in the new era
Pinelopi Anagnostopoulou1, Sandra Rovira-Amigo2,3
1Medical School, University of Cyprus, Nicosia, Cyprus.
ERJ Open Research
|March 25, 2026
Insights
Prospective studies are needed to understand how young children with cystic fibrosis (CF) develop during the new modulator therapy era. This research aims to provide a comprehensive overview of their growth and health outcomes.
Area of Science:
- Pediatric Pulmonology
- Genetic Diseases
- Pharmacology
Background:
- Cystic fibrosis (CF) is a genetic disorder affecting multiple organs.
- CFTR modulator therapies have transformed treatment paradigms.
- Long-term outcomes in young children under these new therapies are not well-documented.
Abstract:
There is a need for prospective studies that provide a comprehensive overview of young children with cystic fibrosis growing up in the modulator era https://bit.ly/4pz0Rqw.
More Related Videos
Related Concept Videos
01:24Cystic Fibrosis: Management
616
Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic...
Sinus disease and chronic...
616
01:23Cystic Fibrosis: Pathogenesis
1.0K
Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
1.0K
01:27Chronic Obstructive Pulmonary Disease-IV: Assessement and Diagnostic Studies
3.4K
Assessing and diagnosing Chronic Obstructive Pulmonary Disease (COPD) involves a detailed approach that includes a comprehensive review of medical history, physical examination, and a variety of diagnostic tests. This thorough evaluation is essential to ensure an accurate diagnosis and guide effective management strategies.
Medical History
Medical History
3.4K