Huntington's disease successfully treated for first time

I've been watching this treatment for a while, in my opinion it's one of the most exciting development in modern medicine. It represents a lot of potential - Huntington's is one of many brain diseases related to protein aggregates, so this technology could be adapted to other diseases. Plus, this is the first curative treatment for what was otherwise a 100% fatal genetic condition.

If anyone ever needs fodder for arguments against people who want to dismantle public healthcare:

Spinraza costs $750,000 in the first year and $375,000 after that in the US, but its manufacturer, Biogen, has agreed a substantial discount with the NHS.

The NHS is smaller than any two of the biggest US health insurers, but got a discount because of its ability to controll the market. This is why it's important that the government be the giant entity making these deals, not private companies.

Private company gets an agreement then charge double base price 👌

Ive been folding @ home and sequencing proteins related to huntingtons research for a while now. I know i dont know much about science, but it makes me happy that maybe my computer helped even a tiny bit to help this venture. and now we can keep on perfecting it knowing its not for nothing.

Even if you didn't work on the winning protein you helped.
Just like a rescue search party you looking in one place let someone else look in another. The person who finds the missing person is random. However if they were working alone they wouldn't have found anything.
- Huh, just like voting.
I thought this was one of silly/joke on every thread but you're being serious. You helped cure them!
We may have never expected to profit but can’t put share of the profit go to reduce that cost to the victim? Even a tiny bit?

The new treatment is a type of gene therapy given during 12 to 18 hours of delicate brain surgery.

...

Treatment is likely to be very expensive. However, this is a moment of real hope in a disease that hits people in their prime and devastates families.

Great news for the scions of billionaires, I guess.

You missed the mark by a mile on this article.
Huntington's disease successfully treated for first time
Read. That. Again.
It was a 18 hours of surgery. The advancement is to be celebrated, not whined about because it's CURRENTLY expensive. People will be saved from decades of agony. You sound like one of those people that claims some secret cabal is hiding the cure for cancer so they can make money.
- I appreciate your positivity. This is a huge breakthrough and means that we'll most likely continue to make this treatment better and more accessible!
  Thats not even mentioning how this treatment may help us figure out how to combat other diseases.
- It will be expensive forever. A similar therapy for SMA children (without surgery) costs US$2.1M. Novartis then gave away doses by lottery. for a few families, they got a dose, for everyone else, their child dies.
  How does Novartis CEO Vasant Narasimhan sleep at night? In a large mansion outside Boston.
- Actually, you've missed the mark. It's not whining about an advancement, it's legitimate criticism of the US health industry. He's just saying what we all know to be true which is that regardless of technological improvements, lifesaving care will continue to be ruinously expensive for those that are able to access it and gatekept from many others.
  If you have a problem with comments like these undermining celebration of scientific progress, then maybe you should think about the structural political issues that lead people to disillusionment and cynicism rather than labeling people as conspiracy theorists.
- The advancement is to be celebrated, not whined about
  It would be celebrated in a country that wasn't clawing back access to health care at every turn
It’s a start.
These things always get better and cheaper with time.
- No they don't. It's not a electronic gadget. They will get off patent in 20 years (this is an orphan drug disease).
- If only. Per capita health care spending has been rising since the 80s, even adjusted for age
There you go. Find that little nugget of negativity in something so positive. Bravo.
New treatment for previously incurable disease? Nah, fuck that. Let's make it about something else that I don't like.
- There is another drug, oral pill, now in phase three. That BBC article was written by UniQure.
Or for people living in sane countries.
It always starts that way. But good God any treatment for a disease the was a guaranteed death sentence is absolutely amazing.
It will cost over US$3M. BBC please stop quoting paid consultants to drug companies without revealing their conflicts.
In a real society, that would get done for everyone with such a rare and severe disease.
- In a real society? What's a real society? Where's this utopia you speak of?

A close friend cares for her father who has it, she could possibly have inhereited it as well. Happy to see I'll have my friend around longer, either way! :)

Very incredible news, that can open so many doors to treating other diseases as well

runs in my family, probably too late so save my brother

Sucks having a disease like this right now. Treatments are so close yet so far away. You get to spend your shortened life hearing about all these advances but none of them will help you because they are too primitive and expensive.

Tylenol STRIKES AGAIN! /s

It's very sad to see hype like this. They only had 30 enrolled, this was just a safety study, and while the data looks promising, there is certainly not enough statistical power, which is why there is no approval yet.

Shame on the BBC. If you are going to quote invesigators, you should state that they have paid consultancy deals with UniQure. The same two people were equally excited about a Roche therapy years ago...

But UniCure stock went up 200% today, which is the point.

Even if they saved a single person it’d be statistically significant.

Today on Mythbusters Kari Byron tests a new Huntington's disease treatment.