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<p>Cystic fibrosis (CF) is a genetic disorder characterized by progressive lung disease and extra-pulmonary manifestations with notable sex disparities in disease outcomes. In this review we summarize the underlying mechanisms driving this sex disparity, with a particular focus on the role of sex hormones on CF lung disease pathophysiology. We explore how the introduction of highly effective modulator therapies (HEMT) may impact sex differences in outcomes and assess whether they have the potential to close the sex gap. While treatment with HEMT has led to better outcomes in the CF population as a whole, females with CF continue to experience worse pulmonary morbidity than males. There is a need for continued research in this area, particularly into the influence and therapeutic potential of sex hormones.</p>

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Sex disparities in cystic fibrosis in the era of highly effective modulator treatment

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年5月2日

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  • Emma McNally,
  • Michelle Casey

摘要

Cystic fibrosis (CF) is a genetic disorder characterized by progressive lung disease and extra-pulmonary manifestations with notable sex disparities in disease outcomes. In this review we summarize the underlying mechanisms driving this sex disparity, with a particular focus on the role of sex hormones on CF lung disease pathophysiology. We explore how the introduction of highly effective modulator therapies (HEMT) may impact sex differences in outcomes and assess whether they have the potential to close the sex gap. While treatment with HEMT has led to better outcomes in the CF population as a whole, females with CF continue to experience worse pulmonary morbidity than males. There is a need for continued research in this area, particularly into the influence and therapeutic potential of sex hormones.