Ivacaftor: a novel gene-based therapeutic approach for cystic fibrosis.
Ivacaftor: a novel gene-based therapeutic approach for cystic fibrosis.
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DOI：
10.5863/1551-6776-18.1.8
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发表时间：
2013-01-01
期刊：
影响因子：
--
通讯作者：
Bradshaw, Marquita D
中科院分区：
文献类型：
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作者：
Condren, Michelle E;Bradshaw, Marquita D
异烟肼是一种新的治疗药物，作用于囊性纤维化跨膜传导调节(CFTR)通道，改变活性。它被批准用于6岁及以上囊性纤维化患者，这些患者在CFTR基因中至少有1个G551D突变。它不同于任何其他目前治疗囊性纤维化的药物，因为它专门针对与囊性纤维化相关的基因缺陷，而不是治疗由此产生的症状。粘液活性药物、抗生素、吸入性β受体激动剂和其他抗炎药目前是囊性纤维化治疗的主要药物，但除了给药频率繁琐外，还可能与几种副作用有关。IVacaftor的口服给药方案提供了更方便的治疗选择。然而，它与显著的药物-药物相互作用有关。
Ivacaftor is a new therapeutic agent that acts at the cystic fibrosis transmembrane conductance regulator (CFTR) channel to alter activity. It is approved for use in patients 6 years and older with cystic fibrosis who have at least 1 G551D mutation in the CFTR gene. It is unlike any other current pharmacologic agent for cystic fibrosis in that it specifically targets the gene defect associated with cystic fibrosis as opposed to treating resulting symptomology. Mucoactive agents, antibiotics, inhaled beta agonists, and other anti-inflammatory agents are currently the mainstay of cystic fibrosis treatment but can be associated with several side effects in addition to cumbersome frequency of administration. Ivacaftor's oral dosing regimen offers a more convenient treatment option. However, it is associated with significant drug-drug interactions.