your limit has been reached. plz Donate us to allow your ip full access, Email: sshnevis@outlook.com
Uptodate Reference Title
Selection of CFTR modulator therapy for patients with cystic fibrosis who are ≥6 years
Selection of CFTR modulator therapy for patients with cystic fibrosis who are ≥6 years
Tables show mutations approved by the US Food and Drug Administration as of January 2021.
CFTR: cystic fibrosis transmembrane conductance regulator; ELX-TEZ-IVA: elexacaftor-tezacaftor-ivacaftor (TRIKAFTA); TEZ-IVA: tezacaftor-ivacaftor (SYMDEKO); IVA: ivacaftor (KALYDECO). * For patients who are not eligible for any available CFTR modulator, consider enrollment in a clinical trial, if available for the patient's genotype. ¶ ELX-TEZ-IVA has not been approved for use in these 5 "splice site" mutations, because the assay used as a predictor of efficacy is not valid for this type of mutation. Clinical studies of ELX-TEZ-IVA have not included sufficient numbers of patients with these rare mutations to assess efficacy in this population. However, the demonstrated efficacy of IVA and TEZ-IVA for patients with these mutations suggests that ELX-TEZ-IVA would also be effective.