Personalized medicine with drugs targeting the underlying protein defect in cystic fibrosis: is monitoring of treatment response necessary?

• Päätekijät: Katharina Niedermayr (Tekijä), Verena Gasser (Tekijä), Claudia Rueckes-Nilges (Tekijä), Dorothea Appelt (Tekijä), Johannes Eder (Tekijä), Teresa Fuchs (Tekijä), Lutz Naehrlich (Tekijä), Helmut Ellemunter (Tekijä)
• Aineistotyyppi: Kirja
• Julkaistu: SAGE Publishing, 2022-07-01T00:00:00Z.
• Aiheet: article
• Linkit: Connect to this object online.

Cystic fibrosis (CF) is caused by two mutations in the Cystic Fibrosis Transmembrane Conductance Regulator ( CFTR ) gene. In the last years, drugs targeting the underlying protein defect like lumacaftor/ivacaftor (LUM/IVA) or tezacaftor/ivacaftor (TEZ/IVA) and more recently elexacaftor/tezacaftor/iv...