REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report | Zendy

Fuchs Teresa | Zendy; Appelt Dorothea | Zendy; Niedermayr Katharina | Zendy; Ellemunter Helmut | Zendy

Open Access

REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report

Author(s) -

Fuchs Teresa,

Appelt Dorothea,

Niedermayr Katharina,

Ellemunter Helmut

Publication year - 2022

Publication title -

clinical case reports

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 0.21

H-Index - 9

ISSN - 2050-0904

DOI - 10.1002/ccr3.5364

Subject(s) - ivacaftor , cystic fibrosis , medicine , newborn screening , sweat , cystic fibrosis transmembrane conductance regulator , pediatrics , gastroenterology

This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible.

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