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Purpose of Review <p>This review aims to synthesize recent advancements in Cystic Fibrosis (CF) treatment, specifically evaluating the triple combination modulator therapy Tezacaftor-Elexacaftor-Ivacaftor. It seeks to compare the efficacy of available modulator therapies, assess the emerging role of gene therapy, and discuss implications for special populations.</p> Recent Findings <p>Clinical trials (TRIKAFTA, VX-445) demonstrate that Tezacaftor-Elexacaftor-Ivacaftor, approved for individuals ≥ 6 years with specific CFTR mutations, significantly improves lung function and quality of life. Safety profiles are generally favorable, though long-term data collection is ongoing. Comparative analyses highlight the superior efficacy of this triple therapy over earlier modulator regimens for eligible patients. Research into gene therapy continues to evolve within the CF treatment landscape.</p> Summary <p>Tezacaftor-Elexacaftor-Ivacaftor represents a transformative advancement in CF management, offering substantial clinical benefits for a large patient subset. Modulator therapies have fundamentally altered disease progression, shifting focus towards highly effective CFTR modulation. Future research must address long-term outcomes, expand access to modulator therapies for all mutation classes, refine gene therapy approaches, and optimize care for special populations in this new treatment era.</p>

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Modulator Therapy: A Narrative Review of the Triple Combination of Tezacaftor, Elexacaftor, and Ivacaftor in the Management of Cystic Fibrosis

  • 期刊论文

  • 发表日期：

    2025年9月4日

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  • Patrick Ashinze,
  • Abdullaah Idris Agbabiaka,
  • Eniola Akande,
  • Aremu Sikiru,
  • Onosegbe Moses Aikhuomogbe,
  • Elelu Taiye Muhammed,
  • Eniola Obafemi,
  • Akogwu Edache Ocholi,
  • Egbunu Emmanuel,
  • Nelson Mafua,
  • Suvam Banerjee

摘要

Purpose of Review

This review aims to synthesize recent advancements in Cystic Fibrosis (CF) treatment, specifically evaluating the triple combination modulator therapy Tezacaftor-Elexacaftor-Ivacaftor. It seeks to compare the efficacy of available modulator therapies, assess the emerging role of gene therapy, and discuss implications for special populations.

Recent Findings

Clinical trials (TRIKAFTA, VX-445) demonstrate that Tezacaftor-Elexacaftor-Ivacaftor, approved for individuals ≥ 6 years with specific CFTR mutations, significantly improves lung function and quality of life. Safety profiles are generally favorable, though long-term data collection is ongoing. Comparative analyses highlight the superior efficacy of this triple therapy over earlier modulator regimens for eligible patients. Research into gene therapy continues to evolve within the CF treatment landscape.

Summary

Tezacaftor-Elexacaftor-Ivacaftor represents a transformative advancement in CF management, offering substantial clinical benefits for a large patient subset. Modulator therapies have fundamentally altered disease progression, shifting focus towards highly effective CFTR modulation. Future research must address long-term outcomes, expand access to modulator therapies for all mutation classes, refine gene therapy approaches, and optimize care for special populations in this new treatment era.