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>Ivacaftor + tezacaftor + elexacaftor (kaftrio0) in cystic fibrosis with at least one F508del mutationRespiratory symptoms alleviated in the shortterm
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Ivacaftor + tezacaftor + elexacaftor (kaftrio0) in cystic fibrosis with at least one F508del mutationRespiratory symptoms alleviated in the shortterm
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机译:Ivacaftor + tezacaftor + elexacaftor (kaftrio0) in cystic fibrosis with at least one F508del mutationRespiratory symptoms alleviated in the shortterm
In patients with cystic fibrosis and at least one F508del mutation in the CFTRgene, triple therapy comprising ivacaftor + tezacaftor + elexacaftor alleviated respiratory symptoms in comparative trials, none of which had a duration of more than 24 weeks. Triple therapy with these drugs was more effective by this measure than the comparator, which was tailored to patients' CFTR mutations. A reduced incidence of pulmonary exacerbations was demonstrated, mainly in a trial in patients heterozygous for the F508del mutation and a mutation resulting in minimal CFTR protein function. The adverse effects of this combination mainly include upper respiratory tract infections, hepatic disorders, rash, muscle disorders. It is also involved in many drug interactions. Any longer-term adverse effects, or the possible effect on disease progression, are not yet known.
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