Today, Vertex applied to the FDA for a drug (Kalydeco) that targets the underlying cause of CF (rather than a drug to treat the symptoms). Here is the full article. Although, Dean doesn't have the G551D change that this drug targets, we're ecstatic for those that will benefit from this and patiently await the results as well as the drug that is in the pipeline that will treat the change (DF508) that he has. Keep those prayers coming, they're working people!!!
http://www.cff.org/aboutCFFoundation/NewsEvents/Vertex-Submits-VX-770-Application-to-FDA.cfm
a different kind of perfect
i'm a mom...on a mission...to enjoy life everyday...appreciate the little moments...try not to get so easily annoyed...raise my kids with a faith legacy that leads them to continue to be amazing people...and to make my husband feel as special as he makes me feel.
What is Cystic Fibrosis?
CF is an inherited chronic disease that affects the lungs and digestive system of about 30k children and adults in the US (70k worldwide). A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that both clogs the lungs and obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food. [www.cff.org]
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