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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).

Matthew HeneghanKevin W SouthernJared MurphyIan P SinhaSarah J Nevitt
Published in: The Cochrane database of systematic reviews (2023)
and QoL. There is probably little or no difference in adverse events for triple therapy (elexacaftor-tezacaftor-ivacaftor/deutivacaftor; VX-659-tezacaftor-ivacaftor/deutivacaftor; VX-440-tezacaftor-ivacaftor; VX-152-tezacaftor-ivacaftor) in pwCF with one or two F508del variants aged 12 years or older (moderate-certainty evidence). Further RCTs are required in children under 12 years and those with more severe lung disease.
Keyphrases
  • cystic fibrosis
  • copy number
  • physical activity
  • genome wide
  • early onset
  • transcription factor
  • cell therapy