A Therapy for Most with Cystic Fibrosis.
A Therapy for Most with Cystic Fibrosis.
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DOI：
10.1016/j.cell.2019.12.032
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发表时间：
2020-01-23
期刊：
影响因子：
64.5
通讯作者：
Bear, Christine E
中科院分区：
文献类型：
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作者：
Bear, Christine E
TRIKAFTA 是 FDA 批准的第三种可挽救主要突变 F508del 引起的缺陷的药物。它优于被批准用于 F508del 纯合子患者的前代产品，因为 TRIKAFTA 对仅携带该突变的一个拷贝的 CF 患者也有效。
TRIKAFTA is the third drug approved by the FDA that rescues defects caused by the major mutation F508del. It is superior to its predecessors that were approved for patients who are homozygous for F508del because TRIKAFTA is also effective in CF patients who harbor only one copy of this mutation.