**Cystic Fibrosis Drug’s Breakthrough for Newborns**
In an exciting medical development, researchers at RCSI University of Medicine and Health Sciences and Children’s Health Ireland have demonstrated the safety and effectiveness of a cystic fibrosis drug in infants as young as four weeks old. This groundbreaking discovery brings hope to families affected by this debilitating condition.
### A Target for Cystic Fibrosis
Cystic fibrosis is an inherited disease affecting the lungs and digestive system. It results from a genetic mutation that impairs the function of a protein responsible for regulating salt and water balance in the body. This malfunction leads to thick, sticky mucus buildup, which clogs the lungs, airways, and other organs.
### The Promise of Ivacaftor
Ivacaftor, also known as Kalydeco, is a drug that targets the underlying genetic defect causing cystic fibrosis. It has been approved for use in adults and children, but its efficacy in newborns had not been previously established. Researchers conducted a study involving young cystic fibrosis patients to investigate this potential.
### A Milestone in Cystic Fibrosis Treatment
The study results published in the Journal of Cystic Fibrosis showed that Ivacaftor was safe and effective in newborns with cystic fibrosis. Prof Paul McNally, one of the study’s authors, hailed the finding as “a huge moment in cystic fibrosis.” He explained that the drug can now be administered immediately upon diagnosis, offering hope and peace of mind to families.
### Earlier Treatment, Better Outcomes
The earlier treatment begins, the better the outcomes for individuals with cystic fibrosis. Starting Ivacaftor at four weeks of age allows for prompt intervention to mitigate disease progression, prevent lung damage, and improve overall health. This is especially significant in Ireland, which has one of the highest incidences of cystic fibrosis globally.
### Impactful Siblings
Two young cystic fibrosis patients, siblings Kara and Isaac Moss, participated in the study. Their mother, Debbie, shared her joy at their remarkable progress. “Kara and Isaac are doing really well and aren’t experiencing any of the typical cystic fibrosis symptoms at the moment,” she said. “These research studies are crucial for ensuring children have access to appropriate treatments as early as possible.”
### Looking to the Future
The RCSI research, supported by Children’s Health Ireland and international collaborators, paves the way for newborns with cystic fibrosis to receive prompt and effective treatment. The pharmaceutical company Vertex Pharmaceuticals, which manufactures Ivacaftor, is seeking an extension of marketing authorization down to one month of age.
### Conclusion
This breakthrough in cystic fibrosis drug treatment for newborns represents a significant step forward in the fight against this challenging condition. Early intervention with Ivacaftor offers hope for a brighter future for affected children, alleviating symptoms, improving their quality of life, and potentially altering the course of the disease.
Deepika 
Mehek 
Divya