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Updated: Jun 17, 2025

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囊性纤维化症是什么

Marcus A Mall1,2,3, Pierre-Régis Burgel4,5, Carlo Castellani6

  • 1Department of Paediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität, Berlin, Germany. marcus.mall@charite.de.

Nature reviews. Disease primers
|August 8, 2024
PubMed
概括

囊性纤维化 (CF) 是一种由CFTR基因突变引起的遗传疾病. 新的CFTR调节器疗法提供了显著的好处,但让许多患者有未满足的医疗需求.

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