Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation

Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation

Background/objectives: Cystic fibrosis (CF) is a life-limiting genetic disorder affecting multiple organ systems. This study compared clinical outcomes, hospitalization rates, and survival between children and adolescents with CF who received CFTR modulator therapies (ivacaftor, lumacaftor, tezacaft...

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Main Authors: Eman A. Toraih, Hassan A. Malik, Rahib K. Islam, Humza A. Pirzadah, Ahmed Abdelmaksoud, Rami M. Elshazli, Paul Antwi Boasiako, Shehab Ahmed Alenazi, Angelique Dabel, Jessan A. Jishu, Bandar T. Alenezi, Hani Aiash, Manal S. Fawzy
Format: Article
Language:English
Published: MDPI AG 2025-01-01
Series:Children
Subjects:
genetic inheritance
targeted therapy
global research network
multicenter
healthcare burden
Online Access:https://www.mdpi.com/2227-9067/12/2/157
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