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

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...

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Main Authors: Yuliya O. Vasenyova (Author), Irina V. Vakhlova (Author), Oleg Yu. Averyanov (Author)
Format: Book
Published: Union of pediatricians of Russia, 2024-09-01T00:00:00Z.
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3rd Floor Main Library

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