Brady is an almost 5 year old little boy with Cystic Fibrosis [genotype G551D and deltaF508]. I have recommended and prescribed Kalydeco for him, to be started as soon as available.
Based on my review of the supportive scientific data, Brady is an ideal candidate for starting the drug. His genotype predicts that it will be efficacious in preventing or slowing development of lung damage in him. Kalydeco is the first drug to treat the root cause of CF by correcting underlying CFTR dysfunction. Every other drug he takes treats only the symptoms. Every day that he spends with dysfunctional CFTR puts him at risk of significant lung deterioration due to CFTR dysfunction, which results in abnormal airway secretions, infection, inflammation, and irreversible damage. With permanent lung damage being the primary ultimate cause of death in cystic fibrosis, PREVENTION of such damage is our #1 goal. Brady so far has mild lung involvement, making early institution of the drug all the more important; it is proven that damage can be prevented by this new drug, but unlikely that damage can be reversed by this or any other drug. Additional benefits of Kalydeco which may accrue include prevention of liver damage and possibly other organ dysfunction, though these benefits are not yet proven.
I look forward to a rapid positive determination in this case, and would be glad to provide more information or to discuss Brady’s case by phone if it would be helpful.
Thank you very much.”