:

Thida Ong1, Bonnie W Ramsey1

  • 1Division of Pulmonary and Sleep Medicine, Department of Pediatrics, University of Washington, Seattle Children's Hospital, Seattle.

JAMA
|June 6, 2023
PubMed
まとめ

胞性線維症 (CF) は遺伝疾患で 世界中で8万9千人以上が罹患しています 新しいCFTR調節療法により,肺機能が著しく改善され,特定のCFTR遺伝子変異を有する患者の発症が軽減されます.

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