• The use of lumacaftor/ivacaftor to treat acute deterioration in paediatric cystic fibrosis

    4 monthes ago - By Paediatric Respiratory Reviews

    Lumacaftor/ivacaftor is a precision medicine targeting the defective cystic fibrosis transmembrane regulator protein in cystic fibrosis patients homozygous for Phe508del genotype. Whilst there is evidence for efficacy in children aged 6-11years who are stable with good lung function, there is little data about the use of this medication for children with acute deterioration in this age group. We describe the use of this drug to treat a child with an unusually severe exacerbation of CF lung disease and review the potential of lumacaftor/ivacaftor as a rescue therapy in the paediatric CF...
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