Real-World Clinical Outcomes of Lumacaftor-Ivacaftor or Tezacaftor-Ivacaftor Therapy in Children With Cystic Fibrosis Homozygous for Phe508del
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This study aims to evaluate the real-world clinical outcomes of dual CFTR modulator therapy (lumacaftor/ivacaftor or tezacaftor/ivacaftor) in children with cystic fibrosis who are homozygous for the Phe508del mutation. The findings indicate a significant reduction in sweat chloride concentration and modest improvements in lung function after one year of treatment, while other health parameters remained stable, supporting the therapy's efficacy and safety in pediatric patients.
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