On Tuesday I heard some incredible news. After years of research, Kalydeco was finally approved by the FDA!! You may wonder "Well what is Kalydeco?" Kalydeco is a tiny pill only for the G551D gene mutation for CF, which affects only about 4% of CF patients. For the G551D mutation, chloride cannot get into the cell walls, which causes the build up of gunk in the lungs and digestive tract. For the DeltaF508 (the gene mutation that affects about 90% of CF patients, including me), the protein does not reach the cell wall. Now that they have completed the Kalydeco study as the pill is finally available for a handful of Cf Patients, they will finally start the study for the DeltaF508 later this year.
Although this pill isn't for me, I was ecstatic to hear the news. The CF patients that can take it have had much higher PFT scores, better digestion, and more. This is a huge step as progress is rolling along :)
This article has a much more detailed explanation:
http://www.huffingtonpost.com/2012/01/31/kalydeco-cystic-fibrosis-cause-drug_n_1244218.html
As Kip from Napoleon Dynamite had sung: "I love technology!"
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