Targeted therapy for cystic fibrosis starts in Russian children at age 2

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Targeted therapy for cystic fibrosis has begun for children aged 2 and older in the Moscow Region, marking a first in Russia. The Cystic Fibrosis Department of the Scientific Research Institute of Childhood has begun treating patients with the drug “Trikafta” in granular form. Children receive the unregistered form of the drug in the Russian Federation thanks to the Circle of Kindness foundation.

In 2025, 39 children aged 2 to 6 years in the Moscow region will begin to receive this therapy. Previously, since 2022, targeted treatment was available only to children over 6 years old.

“As part of the day hospital in the cystic fibrosis department of the Institute, the effectiveness and safety of the drug are carefully monitored. After just 2 weeks of treatment, patients showed a significant decrease in the level of chlorides in sweat to normal and borderline values. All patients who have started receiving the drug will undergo regular follow-up examinations every 3 months,” said Niso Odinaeva, director of the Research Institute.