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Radiological and long-term clinical response to elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease.

Pediatric pulmonology - 1 Aug 2023

Aalbers Bente L, Mohamed Hoesein Firdaus A A, Hofland Regina W, Bronsveld Inez, Kruijswijk Marian A, Schotman Suzan, Slingerland Corlien W, Panhuis Hannah, van der Ent C Kors, Heijerman Harry G M

Abstract excerpt

INTRODUCTION: A triple combination of CFTR modulators ELE/TEZ/IVA (elexacaftor/tezacaftor/ivacaftor, Trikafta™) has been evaluated in clinical trials for people with cystic fibrosis (pwCF) and was approved to the European and US market. During registration and settling reimbursement in Europe, it could be requested on a compassionate use basis, for patients with advanced lung disease (ppFEV1 < 40). AIM: The aim...

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PMID: 37222401
DOI: 10.1002/ppul.26486

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