I recently wrote a review of Sarepta Pharmaceuticals drug eteplirsen for the treatment of Duchenne muscular dystrophy. The gist of it is that the FDA is about to review the drug and vote whether to approve or reject it, on April 26. There is no significant proof that the drug works. Evidence suggests that it is safe though. Just to get the disclosure out of the way for legal reasons, I own no stake in Sarepta and don’t plan on buying any.
Muscular dystrophy is a horrible horrible disease. I wouldn’t wish it on anybody except members of Congress and the Knesset, but since it’s genetic that’s only rhetorical.
People who have it will die by the time they are 25 or so. Their muscles will decay and they will not be able to move, and then eventually they will not be able to breathe and that’s it.
Eteplirsen tries to repair the broken gene that codes for dystrophin, the protein that most of us have that enables us to move. It might work, it might not. It’s hard to tell. But without it, everyone with muscular dystrophy on exon 54 will die. With it, maybe they will, and maybe they won’t.
The meeting takes place on April 26. There will be record attendance at the meeting, I just read. 1,200 people will watch a group of government doctors determine if dying people will be allowed to take eteplirsen. The entire addressable market for this form of the disease is about 1,560 in the US.
The entire question is absolutely insane. If you don’t realize that, you are amoral, or immoral, or both. If these people want to take the damn drug, they should be able to take it at the agreed upon price, even if it were extremely dangerous, which it isn’t.
The thought of self righteous government scientists who do not have muscular dystrophy deciding for a group of people whether or not they can try the drug, just drives me mad. It makes me want to cry for them.
They will probably reject approval. For the greater good of course. Because if they are God forbid allowed to even try a drug that has no proven efficacy, the entire reason d’etre of the FDA will cease to exist.
Newsflash. There is no reason for the FDA to exist. All they do is kill people in the name of safety.
The Jewish Libertarian 
Anyone with a clue on healthcare in the US knows that the FDA is partially funded by pharmaceutical companies – 40%. Which is a massive and tragic conflict of interest. A federal government agency entrusted with safeguarding the health of the American people partially funded by the private sector is a recipe for disaster. This explains why Americans’ pay by far the most money for their healthcare in the world, yet their healthcare system is ranked 23rd in the world. News update: the FDA’s “Standard of Care” is public enemy number one in America not just for muscular dystrophy but for dozens of illnesses including cancer and Alzheimers disease. In the US, healthcare is a big money game not a healing art as its supposed to be. For example, the active suppression of the deleterious effects of toxic mold robbed me of decades of my life, yet the FDA in alignment with the CDC continues to say that toxic mold merely causes “temporary respiratory irritation” yet it causes me chronic fatigue syndrome. Until enough Americans wake up to the abject corruption of the FDA colluding with big pharma (and the CDC), they and the pharmaceutical companies will continue to exploit uninformed Americans of tens of billions of dollars for lackluster and often toxic “healthcare”. For the desperate, I would begin with a combination of PEMF therapy (magnetic therapy), IV ozone, a USDA organic diet, and vaccine detox homeopathic treatment for muscular dystrophy and begin seeing a good naturalopathic physician for ongoing treatment.
off topic.
what do you think about the economics of this article on local currency?
http://virtualjudah.wordpress.com/2010/10/26/avodah-ivrit-and-economic-independence/
thanks.
It’s complete economic ignorance. This article is filled with stupidity. Perhaps I’ll write a post responding to it. I don’t know this guy but I doubt we would get along. On the power vs liberty spectrum, he’s an extreme power guy.
He is certainly an “extreme power guy” in personality, but he’s a lot worse than that. He wants to mimic the Hamas to force the government to grant settlers an independent ‘Jewish’ state through armed revolution.
But what interests me is not the political part, but the autarkic, mercantilist economics. Is there a better way to economically secede without harming yourself? Is local currency ever viable? How would you suggest we go about seceding effectively?
I’m not a ground up libertarian specialist guy. I’m more of a top down guy, which is why I’m a Feiglin supporter. Not that ground up isn’t necessary – it is, it’s just not my area of expertise. Any time you secede you are foregoing the division of labor. If you know how to do everything yourself then go for it, but most of us need to trade to survive. So the best way to secede economically is to somehow become an expert in providing yourself with everything you need, but that will make a very unproductive life.
If this guy wants to wall himself off economically, that’s fine, he’ll just lose any influence he has, which is not much.