As many of you know, I was diagnosed with cystic fibrosis in 1984 at the age of 3 years old, but what many of you may not know is that when my parent were given that diagnosis they were also told that I would most likely not see my 12th birthday. You see at that time there were no drugs or antibiotics specifically designed to help manage the symptoms of cf, there were no modulators to treat the underlying defect at a cellular level. In fact, it would don't be until 6 years later that Foundation-supported scientists would discover defective CFTR gene that causes cf!
In 30 short years since the discovery of the CFTR gene, tremendous advancements have been made in not only treating the symptoms of cf but also in actually treating the underlying defect caused by the gene. In 2012 the first of these drugs designed to treat the actual defect in the gene, called CFTR modulators, was approved by the FDA. This drug was known as Kalydeco and over the next 6 years we would see the approval of two additional drugs, Orkambi and Symdeko. Most impressively, at the end of last year, we saw the approval the first triple combination therapy, known as Trikafta! This drug could eventually help approximately 90% of those living with cf. Imagine that, in 30 years we have gone from nothing to a drug that corrects the underlying defect in the gene for upwards of roughly 90% of those living with cf.
These kind of advancements are not possible without you and I hope you celebrate these remarkable achievements with us! But we are not done yet, Trikafta is not a cure and it does not work for everyone.
It is with a full and grateful heart that I thank you for your continued love and support and for your kindness and compassion. It is because of each and everyone of you that I am here today and why I am confident that one day, together, we will all be able to say that we cured cystic fibrosis.
Please stay safe and healthy and most importantly continue to BREATHE IN HOPE.