Cystic Fibrosis Breakthrough: Ivacaftor Safe for Newborns
Cystic fibrosis (CF) is a debilitating genetic condition that affects the lungs, digestive system, and other organs. Until recently, there was no cure for CF, but a new drug called Ivacaftor has shown promising results in treating the condition. In a recent groundbreaking study, researchers have found that Ivacaftor is safe and effective for use in newborns as young as one month old.
Understanding Ivacaftor
Ivacaftor, also known by the brand name Kalydeco, is a medication that targets the underlying cause of CF. CF is caused by a mutation in the CFTR gene, which leads to the production of a defective protein that impairs the function of the lungs, digestive system, and other organs. Ivacaftor works by correcting the function of the defective protein, thereby improving the symptoms of CF.
Safety and Efficacy in Newborns
The recent study included 7 babies between the ages of 1 and 3 months who were diagnosed with CF. The babies were given Ivacaftor for a period of 48 weeks, and the results were promising. The babies tolerated the medication well, and there were no serious side effects reported. Additionally, the babies showed significant improvements in their lung function and overall health.
Hope for Families
The findings of this study provide hope for families with newborns who have been diagnosed with CF. Ivacaftor is the first medication that has been shown to be safe and effective for use in newborns with CF. This means that babies can now start treatment immediately after diagnosis, potentially preventing or reducing the severity of the symptoms of CF.
Next Steps
The researchers are currently applying to the European Medicines Agency for an extension to the marketing authorization for Ivacaftor down to one month of age. If approved, this would make Ivacaftor available to an even wider range of newborns with CF.
Conclusion
The discovery that Ivacaftor is safe and effective for use in newborns with CF is a major breakthrough in the treatment of this debilitating condition. This finding provides hope for families with newborns who have been diagnosed with CF, and it could potentially lead to improved outcomes for these children in the future.
also read:When Can Newborns with Cystic Fibrosis Start Treatment with Ivacaftor?