Article
The New and Advanced Cystic fibrosis transmembrane conductance regulators Modulators in Children with Cystic Fibrosis: A Narrative Review on Long-Term Outcomes, Recent Advances, and Comparative Insights.
2025-06-23
Abstract excerpt
Cystic fibrosis (CF) is a progressive, life-limiting genetic disorder caused by mutations in the CFTR gene, leading to dysfunctional chloride channels and multisystem involvement. The introduction of CFTR modulators has revolutionized CF management by targeting the underlying protein defect rather than just alleviating symptoms. These therapies such as Ivacaftor, Lumacaftor/Ivacaftor, Tezacaftor/Ivacaftor, and Tri...
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Identifiers and source
- Literature Corpus work
- a1bde5b9-568f-5ae7-90cc-c8a8d53a5035
- DOI
- 10.22541/au.175065678.87130337/v1
