FDA APPROVES CYSTIC FIBROSIS DRUG!
The novel drug ivacaftor (Kalydeco), formerly known as VX-770, has been approved for treating cystic fibrosis arising from the G551D-CFTR mutation, the FDA said Tuesday.
The drug is the first approved agent that targets abnormal chloride trafficking in cystic fibrosis, the condition’s underlying cause.
Found in 4% to 5% of cystic fibrosis patients, G551D-CFTR is the second most common genetic mutation after the delta F508-CFTR mutation, which is present in more than 90% of patients. An estimated 70,000 patients worldwide and 30,000 in the U.S. have cystic fibrosis.
Ivacaftor increases the time that activated CFTR channels at the cell surface remain open, allowing for more normal chloride flux in lung tissue.
The drug was also associated with greater reductions in pulmonary exacerbations and respiratory symptoms, as well as better weight gain in the study. Patients with cystic fibrosis often have difficulty maintaining normal weight and are excessively thin
This is great, Cystic Fibrosis is such a terrible disease, and were just that much closer to curing it.
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etondreamer reblogged this from sebastaniel and added:
A drug to combat CF?! Yay!
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OH GOD, IS THAT SOMEWHERE INSIDE ME!?
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callingfromtheunderground reblogged this from taylordontcare and added:
if this is legit, well what in the fuck.
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I remember reading about this...a while ago, I can’t believe how quickly this approval...
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This makes my shitty day better no matter what else happens.
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