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تعداد آیتم قابل مشاهده باقیمانده : 3 مورد
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Selection of CFTR modulator therapy for patients with cystic fibrosis who are ≥2 years

Selection of CFTR modulator therapy for patients with cystic fibrosis who are ≥2 years
Tables show mutations approved by the US Food and Drug Administration as of May 2023.

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.
Data from:
  1. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor; ivacaftor) Tablets and Oral Granules. Vertex Pharmaceuticals Incorporated. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217660s000lbl.pdf (Accessed on May 4, 2023).
  2. SYMDEKO® (tezacaftor/ivacaftor; ivacaftor) Tablets. Vertex Pharmaceuticals Incorporated. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/210491s007lbl.pdf (Accessed on December 20, 2020).
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