Cystic Fibrosis Gene Correction Drug Approved by the FDA 264
tguyton writes "The good news: the FDA just approved the distribution of the first drug to treat the underlying cause of Cystic Fibrosis, called Kalydeco by Vertex Pharmaceuticals. The bad news: this drug will only affect 4% of patients with the disease in the U.S. From the article: '[Affected patients] with the so-called G551D mutation have a defective protein that fails to balance the flow of chloride and water across the cell wall, leading to the buildup of internal mucus. The vast majority of cystic fibrosis patients have a different genetic defect, in which the protein does not reach the cell wall. Vertex is developing another drug to try and address that problem. Study data for that drug is expected later this year.' Hopefully the research involved will be applicable to finding treatments for other genetic diseases."
Further bad news: "...executives said Kalydeco would cost $294,000 for a year's supply, placing it among the most expensive prescription drugs sold in the U.S."
Re:So, treating 4000 people (Score:2, Funny)
Oh, stop. The Party line here is to bash on big evil pharma until all drugs are produced for free by unicorns from fields of biodiverse flora.
Turn in your geek badge. You're going all mainstream with your facts and reality and pragmatism.
Re:So, treating 4000 people (Score:4, Funny)
Thank the Flying Spaghetti Monster and his noodly appendages, I live in Canada.
Hopefully the researchers in the evil ignorant US will share what they've learned with the enlightened Canadians.
Re:Cell MEMBRANE (Score:5, Funny)
Good lord, we are animals not plants. There is no such thing as a "cell wall" in our cells! Call it what it is: the cell membrane.
Pedantic? Yes, but the definitions are precise and are intended to be used precisely. Journalism like this makes me want to gouge my eyes out; a single high-school biology class teaches cell wall vs. cell membrane!
I'm a scientist., I'll handle this!
By the power vested in me by science, I hereby retroactively flunk the original submitter's high school biology grades, and also raise the grade of the bookish, socially awkward lab partner you conned into doing all your work. The sentence is to correct 10 obnoxiously factually incorrect slashdot comments without invoking any of the following: Godwin's Law, correlation vs. causation, Ron Paul, or conspiracy theories of any kind. Oh, and just for good measure, rule 34.
Until then your slashdot submitting privileges are subject to double-secret probation.