Alex Barloewen

 State of Cystic Fibrosis Research 2024

Enormous progress has been made in the past 10 years. In 2012, the first drug, Ivacaftor was introduced to treat the symptoms of Cystic Fibrosis. However that drug only applied to 4% of the CF population. In 2019, another drug, Trikafta received FDA approval and expanded treatment to about 85 % of the CF population. This drug has now also been approved for treatment of all 2+ year-old children. The sooner children can be treated with Trikafta the less damage their lungs will incur. The overall impact of these medications has been astounding - in the past 5 years the median predicted survival age for newborn infants has increased from 38 to 56+ years.  60 % of young adults now have normal lung function versus 30 % a decade ago. This is unprecedented!

Our Granddaughter, Abby, has been taking Trikafta, since December 2019  The results have been very positive. During the first few months her lung test results rapidly improved back to normal and have remained at that level for the past 53 months. In addition, she has experienced no hospitalizations due to pulmonary lung exacerbations since she has been taking Trikafta.  She has gained weight and her energy level is much improved. Due to Trikafta she has experience the healthiest years of her life.