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

• Main Authors: Katharina Niedermayr (Author), Verena Gasser (Author), Claudia Rueckes-Nilges (Author), Dorothea Appelt (Author), Johannes Eder (Author), Teresa Fuchs (Author), Lutz Naehrlich (Author), Helmut Ellemunter (Author)
• Formato: Livro
• Publicado em: SAGE Publishing, 2022-07-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: 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...