[karmelapple]

What part of this discovery was made thanks to NIH and/or NSF funding from the USA, or the NIHR in the UK?

I don't ask to strictly bring up politics, but instead to try and address the broad lack of understanding of how medical breakthroughs like this are made.

It's not done just by drug companies. The article says:

> UniQure says it will apply for a licence in the US in the first quarter of 2026 with the aim of launching the drug later that year.

That's true, but that doesn't talk about the tens to hundreds of research papers that have been published over likely decades to make this discovery a reality. And it doesn't talk about how much public money went into this discovery.

Many people reading this article probably have a vague idea that more than just this company was involved, but I feel it is not at all clear to the vast majority of people, since the vast majority of people are not involved in biomedical research.

I wish there was an easy way to figure out how many dollars, how many grants, how many researchers, went into achieving this breakthrough. And that the media would put that into news articles like this. Trace all the citations back a few orders, and I bet you'll find a massive number of NIH and NIHR grants.

There is unfortunately not more massive, bipartisan public outcry in the US over defunding the essential basic research the NIH does... and it's not new to the current administration, since it was attempted to be done back in 2017, too [1].

Scientists need better messaging or else we're going to stop having breakthroughs like this... and the breakthroughs are already going to slow down thanks to things like the $783 million in cuts to NIH grants that the US SCOTUS authorized in August [2].

1. https://pmc.ncbi.nlm.nih.gov/articles/PMC5468112/

2. https://www.scotusblog.com/2025/08/supreme-court-allows-trum...

[stevenwoo]

The problem is the soundbite of some of these studies on the surface is ridiculous to lay people but even good studies with bad sound bites are used as weapons against science funding in the USA. The shrimp on a treadmill study is still used as argument against science funding today. https://www.npr.org/2011/08/23/139852035/shrimp-on-a-treadmi...

[privatelypublic]

I always thought the shrimp was a random meme. This is even better!

My understanding is There's also studies of "duh!" Things, so theres a paper to cite instead of an assumption.

[jmcgough]

> Scientists need better messaging or else we're going to stop having breakthroughs like this

Sure, but it's really sad that scientists need to justify their funding to the public - they already spend so much time justifying it to the NIH and others for funding.

So many people have had their careers jeopardized by finding pulled mid-project. I am really concerned about our research pipeline, because my post-doc friends are all applying to jobs outside the US now.

[mattkrause]

This is where the funders really ought to step up.

A spokesperson from (say) NINDS really ought to be shouting to anyone who will listen about how excited they are to see their <many year>, <many dollar> investment in Huntington's pay off.

I'd love it if they highlighted some of the especially "weird" studies that went into this to demonstrate how important fundamental research is and how it goes in unexpected directions.

[philipallstar]

> it's really sad that scientists need to justify their funding to the public

The (mostly American) public pays their salaries; it's not that odd. The public elect the government (mostly the US government) that distributes the funds. The US voter in general has just got tired of living in the "worst place in the world" while also funding the majority of the world's science and health breakthroughs, I think.

[slfnflctd]

This take seems a tad myopic to me.

We are rapidly and increasingly falling behind at least two other nations in terms of science and health breakthroughs.

The things that make the US seem like the worst place in the world are mostly a result of political gridlock, which is advantageous (in the short term) for a small group of people who thrive under the status quo, who do everything possible with their outsized power to manipulate the electorate to keep it that way, out of cowardice.

The US voter in general has been horribly misinformed, misled and manipulated into supporting things that are both against their own personal interest and against the greater good for the country as a whole.

I don't know how to fix this, but I personally don't think making grand proclamations about how great we are/were while pointing blame fingers around to obfuscate the more obvious reasons for the average citizen's discontent is a smart or ethical way to approach the situation.

[philipallstar]

> We are rapidly and increasingly falling behind at least two other nations in terms of science and health breakthroughs.

Perhaps you are, but I'm not convinced. What's the metric?

> The things that make the US seem like the worst place in the world are mostly a result of political gridlock, which is advantageous (in the short term) for a small group of people who thrive under the status quo, who do everything possible with their outsized power to manipulate the electorate to keep it that way, out of cowardice.

The political lines are clear. You can see endless interviews with kids on college campuses who've been indoctrinated to believe the silliest things. That America is the worst, most racist and sexist place in the whole world.

> The US voter in general has been horribly misinformed, misled and manipulated into supporting things that are both against their own personal interest and against the greater good for the country as a whole.

This seems a tad myopic itself. While I agree people are being misled, I would count both you and I as "people" in that regard, and equally not knowing what's best for everyone. If we did, we could just be the only two voters and it would all be perfect.

> I don't know how to fix this, but I personally don't think making grand proclamations about how great we are/were while pointing blame fingers around to obfuscate the more obvious reasons for the average citizen's discontent is a smart or ethical way to approach the situation.

No idea what this means, as there were no grand proclamations previously mentioned.

I think part of the problem is America can do huge numbers of things right, and carry quite a lot of the advances that help the whole world on its taxpayers' backs, but still be mostly criticised and teased as a country from the citizens of other, generally older, now less high achieving countries. Who only acknowledge those massive, ground-breaking contributions when they are withdrawn and can be used to complain about the USA.

[melagonster]

We even do not need to calculate NIH grants; I am pretty sure that all databases that were used here are from NCBI. If there were no NIH, all research would be impossible in modern biology.

[oulipo2]

I'm pretty sure some of it is direct funding by governmental agencies, but even if that wasn't the case, all the basis of the theory, and the groundwork was laid by researchers and universities using those grants. You need public money for a healthy society

[caycep]

I think it is a UK study. It actually has been going on for a number of years, I've seen one of the PIs giving a number of big conference talks (Tabrizi) for a while

[sleight42]

There's little money to be made with HD. It's a 1 in 30,000 disease. There's been little reason for anyone other than state sponsors to support its treatment. Add this to the reason's to be disgusted by capitalism. Spoken as a widower of an HD wife.

[mnw21cam]

I do research into neonatal diabetes, which is a 1 in 100,000 genetic disease. We're entirely state and charity funded. We have had a grant for many years to do genetic analysis on anyone from around the world who fits the criteria (diabetes under six months age) and who can send in a blood or DNA sample. It's a good model, and now more than 90% of patients with neonatal diabetes get their genetic diagnosis.

[sleight42]

I am VERY glad to hear this. And thank you for your work.

[missedthecue]

I'm sorry to hear about the passing of your wife.

How common a disease is doesn't have much to do with treatment efforts. Cystic Fibrosis has practically been cured by Big Pharma and only ~40,000 people in the entire US have it.

[pkaye]

That is because the Cystic Fibrosis foundation funded some of the research and drug development to make the first treatment possible. They had to essentially operate in a VC model to get the treatments that these CF patients need.

https://www.cff.org/about-us/our-venture-philanthropy-model

[missedthecue]

Most disease categories have advocacy and funding groups, with varying amounts of success. The point is that rare diseases get new treatments all the time from private for-profit firms. In fact, orphan diseases are extremely profitable, and the FDA offers fast tracked approval decisions, making them an enticing niche to focus on.

[tirant]

I'm sorry for your loss, I can only imagine how difficult it must feel to face a disease with so few treatment options

I just would like to say that it's not capitalism that decides if money is invested in a disease or another but just the individuals operating freely in the market. On the other hand Capitalism has been actually the main driver for the massive investments that enable the expensive research in biomedical topics.

It’s unfortunately normal that conditions with very low prevalence, receive less private investment than diseases that affect millions of people. That’s not because of a moral failing of capitalism, but a result of free market and the free decision of the population on where to allocate their resources. Imposing anything else on people would actually be the real moral failing, because what is the right allocation of resources between technological development, investments about hunger, medical development or just leisure? Let each individual decide for themselves and of course feel free to convince anyone to invest in what you consider priority.

So I think the fairest system is the one where individuals remain free to choose how to invest their time and money, while society as a whole can still decide, for example through philanthropy, to give extra support to areas some areas like rare diseases.

Said that, if you know of any organization supporting HD research that deserves any type of donation please let us know here so we can support it voluntarily.

[kulahan]

It really has nothing to do with capitalism. There are special grants in the US for researching rare diseases, specifically to ensure money isn't the barrier.

As an aside because I'm pedantic about the language, apostrophes are never used to show pluralism.

[kens]

> As an aside because I'm pedantic about the language, apostrophes are never used to show pluralism.

Mind your p's and q's.

Source: Chicago Manual of Style, 14th edition, section 6.82. Also see section 6.77: Tim had had enough of her "maybe's."

[kulahan]

I am unironically stunned

[karmelapple]

I am scared that special grants to research rare diseases will go away, too.

If we're trying to figure out what the most benefit for each taxpayer dollar is, then a rare disease won't win out over, say, cancer research.

Someone may consider researching a rare disease as "waste," even though to everyone including the previous poster who is a widow because of HD, it is far from a waste.

When there is not much of a profit motive to do something - whether going to the moon or fighting a rare disease - public money is the best way to do it. And even throwing a fairly small percentage at it can create big achievements.

And that's one reason I'd like to see how much money and time went into this. We might be surprised that it's fairly small in the grand scheme of biomedical research costs!

[jjtheblunt]

> apostrophes are never used to show pluralism.

in Dutch they are for some words, e.g., 1 ski, 2 ski's. i have no idea how that arose historically.

[kulahan]

The Nords are a pox upon us all. TIL though, thanks - I probably should've been clear I only meant in English :)

[J_McQuade]

If you think the Dutch are Nords, then I would ask you to learn more English.

[amelius]

It's standing on the shoulders of giants, and the one on top gets to reap all the benefits.

[mattkrause]

Even that quote is a bit of a disservice to modern science: it's a massive pyramid made of thousands and thousands of individual contributions, including many bits of deep background and outright "failures".

Biology is tough in that you can't just "reason" your way to success; it often really does require trying something to see if an approach works.