Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis.

Pavel Drevinek,Tacjana Pressler,Marco Cipolli,Kris De Boeck,Carsten Schwarz,Florilene Bouisset,Marie Boff,Noreen Henig,Nicolas Paquette-Lamontagne, Sonya Montgomery,Jaakko Perquin,Nigel Tomkinson,Wilhelmina den Hollander,J Stuart Elborn

Journal of Cystic Fibrosis（2020）

引用 46|浏览23

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摘要

•Eluforsen was safe and well tolerated in F508del-CFTR homozygous CF patients.•Single and multiple doses of inhaled eluforsen showed low systemic exposure.•Multiple doses of inhaled eluforsen improved CFQ-R RSS (exploratory endpoint).•Results support further development of eluforsen for CF F508del-CFTR homozygous patients.

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关键词

Antisense oligonucleotide,Clinical trial,Delta F508,CFQ-R RSS,Pulmonary medicine

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