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A new era of cystic fibrosis therapy with CFTR modulators.

作者信息

Barros Laura Gomes Boabaid de, Prestes Laura Menestrino, Fernandes Maria Fernanda Gonçalves Meirelles, Silva-Filho Luiz Vicente Ribeiro Ferreira da, Pinto Leonardo Araújo

机构信息

. Grupo de Pesquisa em Epidemiologia e Genética das Doenças Respiratórias da Infância, Pontifícia Universidade Católica do Rio Grande do Sul - PUCRS - Porto Alegre (RS) Brasil.

. Instituto da Criança e do Adolescente, Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo, São Paulo (SP) Brasil.

出版信息

J Bras Pneumol. 2025 Jan 20;50(6):e20240405. doi: 10.36416/1806-3756/e20240405.

DOI:10.36416/1806-3756/e20240405
PMID:39841779
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11796393/
Abstract
摘要

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A new era of cystic fibrosis therapy with CFTR modulators.使用CFTR调节剂治疗囊性纤维化的新时代。
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Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis.囊性纤维化 CFTR 变异体特异性治疗(包括调节剂)的护理标准。
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本文引用的文献

1
Cystic fibrosis.囊性纤维化。
Nat Rev Dis Primers. 2024 Aug 8;10(1):53. doi: 10.1038/s41572-024-00538-6.
2
Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.使用 elexacaftor+tezacaftor+ivacaftor 治疗至少携带一个 F508del 等位基因的囊性纤维化个体:系统评价和荟萃分析。
J Bras Pneumol. 2024 Jan 5;49(6):e20230187. doi: 10.36416/1806-3756/e20230187. eCollection 2024.
3
Cystic Fibrosis: A Review.囊性纤维化:综述。
JAMA. 2023 Jun 6;329(21):1859-1871. doi: 10.1001/jama.2023.8120.
4
Worldwide rates of diagnosis and effective treatment for cystic fibrosis.全球囊性纤维化的诊断和有效治疗率。
J Cyst Fibros. 2022 May;21(3):456-462. doi: 10.1016/j.jcf.2022.01.009. Epub 2022 Feb 4.