Evaluation of the Effectiveness of Using the CFTR Modulator Ivacaftor/Lumacaftor in Children with Cystic Fibrosis in the Sverdlovsk Region (Prospective Cohort Study)

• 主要な著者: Yuliya O. Vasenyova (著者), Irina V. Vakhlova (著者), Oleg Yu. Averyanov (著者)
• フォーマット: 図書
• 出版事項: Union of pediatricians of Russia, 2024-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

Background. A modern achievement in the treatment of cystic fibrosis has been the discovery of small molecules that restore the processes of synthesis, transport to the membrane, and the work of the defective CFTR protein. Drugs whose action is aimed at restoring the function of the CFTR protein are...