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Article

Trials and tribulations of highly effective modulator therapies in cystic fibrosis.

Paediatric respiratory reviews - 1 Dec 2023

Lieu Nathan, Prentice Bernadette J, Field Penelope, Fitzgerald Dominic A

Abstract excerpt

Highly effective modulator therapies (HEMTs) have revolutionised the management approach of most patients living with cystic fibrosis (CF) who have access to these therapies. Clinical trials have reported significant improvements across multiorgan systems, with patients surviving longer. However, there are accumulating case reports and observational data describing various adverse events following initiation of...

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PMID: 37914566
DOI: 10.1016/j.prrv.2023.09.001

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