Highly Effective Modulator Therapy in Cystic Fibrosis: Addressing Unusual Variants in the Middle East.

Abstract excerpt

Background: Cystic fibrosis (CF) is an autosomal recessive disorder caused by variants in the CFTR gene. Although the F508del mutation is common globally, the Middle East exhibits a higher prevalence of rare, region-specific variants. The triple-combination therapy elexacaftor/tezacaftor/ivacaftor (ETI) has revolutionized CF management; however, its efficacy in individuals with rare variants, often...

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