The radio stations are filled with holiday music, but as 2015 comes to a close, I find myself listening to an entirely different playlist. In my mind, there are not only songs that make me reflect on the path that my family has so carefully traveled so far, but also the hope and the promise of all the good things yet to come.
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The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
CF Foundation's $1 Million Investment Helped Drug Known as Cayston® Become a Reality
New Effort Will Search for Potential Therapies Targeting the Most Common Mutation of Cystic Fibrosis
Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
I’ve learned that paying too much attention to the idealized lives of people on social media can give me a skewed vision of my own.
At age 5, my daughter started to feel the traumatic emotional effects of cystic fibrosis. Fortunately, the care center’s social worker helped her find a healthy path through it.
I wish people knew that even if I don’t look sick, I still struggle with the mental and physical aspects of cystic fibrosis. Even my family and friends don’t truly understand what this disease does to your mental health.
This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.