Kaftrio Treated Severe CF Without Confirmed Secondary CFTR Mutation

Kaftrio Treated Severe CF Without Confirmed Secondary CFTR Mutation

302021

Kaftrio Treated Severe CF Without Confirmed Secondary CFTR Mutation

Kaftrio successfully improved lung function and quality of life in three Italian cystic fibrosis (CF) patients who have severe lung disease with an unknown mutation in the CFTR gene in addition to the common F508del mutation, a case series reported. Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the U.S. and marketed by Vertex Pharmaceuticals, is available only for CF patients with at least one CFTR F508del mutation or another known secondary mutation. Patients whose DNA…

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