Cystic Fibrosis is a complex and sometimes confusing disease. Each installment of Breathe 3-65 called,"Simply CF" will strive to explain the complexities of CF in a concise and accessible way.
While a life with cystic fibrosis may have its share of days that are difficult and burdensome, there are indeed days that are filled with unprecedented celebration in our CF community. With continued hope there surely will be many more days of celebration to come.
This past week on Thursday, September 29 there was cause for great celebration in the world of CF. Orkambi, a specialty medication that treats the underlying cause of cystic fibrosis for people with two mutations of F508del, was approved by the FDA for children as young as six. This news gives such hope to so many families and people with CF. In July of 2015 Orkambi was only approved for people with cystic fibrosis twelve years of age and older.
Orkambi and Kalydeco
There are now two life changing drugs of its type in the fight against CF: Orkambi and Kalydeco. These medications are the first of their kind approved by the FDA in the CF community. These medications do not treat the symptoms of CF but target the underlying cause of CF down to the genes themselves. These medications aim to improve the functionality of the CFTR proteins. They aim to make the mutated genes work more normally in hopes that the progressive debilitating effects of cystic fibrosis will be slowed or minimized. This is not a cure and there’s no guarantee that all who take it will experience a great margin of positive change. But it is surely life changing and something to greatly celebrate.
Within the last twenty-five years there have been incredible advances in the treatment of CF. Advances that have allowed many of us to live well beyond that in which our parents were told. Advances that allow every day to be a celebration for those of us living with CF. Advances that give us hope that someday all people with CF regardless of their mutations will be eligible for such life-changing drugs. Advances that will give us all another day to celebrate together.