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Huntington's disease treated for first time

bbc.com

31–40 of 133 posts

Re: Huntington's disease treated for first time

#31
post #28
post #21

Medical progress has been insane in the last few years through technological breakthroughs. It's not out of reach to think that most types of cancers will be curable 20 years from now on.

[flagged]

This isn’t the place for that.

Yes, the autism+Tylenol thing is infuriating, but there are nobler ways to express that opinion.

Re: Huntington's disease treated for first time

#32
post #24

This is off topic, slightly but I think a good place to say this: I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen) In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders. Just a thought.

This seems helpful, but I think the misattribution of a general "anti-immigrant" sentiment to immigration detractors is part of the problem.

Very few detractors in the west have any issues with highly qualified immigrants occupying scientific or research roles. Being opportunistic with which kind of immigrants one offers as Good is partly what's aggravating the issue. It's a radical kind of dismissiveness and denialism which is provoking people and ignoring their issues.

The broad western detraction against immigration at the moment is targeted at specific waves of mass immigration with specific compositions that have specific effects on the places those immigrants have landed.

People are primarily concerned about the ability of state, social and corporate institutions to absorb immigrants at this pace and scale without significant zero-sum effects. And, in addition, the significant amount of state support segments of those populations (eg., esp. asylum seekers) have to receive at a time when gov. are under inflationary pressures, debt pressures, etc. and cannot service their own welfare obligations.

Going, "oh but we get good cancer research from immigration!" is so dismissive to these concerns, that the backfire against this messaging is one of the major contributors to people's disaffection.

The idea that people need to be told that there are people who want to immigrate that are in our national interest to absorb, is just plainly absurd. This is uncontroversial and obvious.

Re: Huntington's disease treated for first time

#33
post #24

This is off topic, slightly but I think a good place to say this: I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen) In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders. Just a thought.

I strongly disagree with this. While I am generally pro-immigration, injecting a political view into an article ostensibly about a new scientific discovery is how science loses credibility and objectivity. See the "trust the science" phrase weaponized during COVID in the US.

Let people draw all the inferences they want about the origins of the scientists involved, but a hamfisted paragraph about a.b scientist being an immigrant from y country does not have a place here.

Re: Huntington's disease treated for first time

#34
post #33
post #24

This is off topic, slightly but I think a good place to say this: I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen) In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders. Just a thought.

I strongly disagree with this. While I am generally pro-immigration, injecting a political view into an article ostensibly about a new scientific discovery is how science loses credibility and objectivity. See the "trust the science" phrase weaponized during COVID in the US. Let people draw all the inferences they want about the origins of the scientists involved, but a hamfisted paragraph about a.b scientist being a…

Also it sends a message that only scientists are welcome as immigrants. There's millions of immigrants who contribute positively to the society, who aren't scientists.

Re: Huntington's disease treated for first time

#36
post #18

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 laun…

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

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.

Re: Huntington's disease treated for first time

#37

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 laun…

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.

Re: Huntington's disease treated for first time

#38

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 laun…

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.

Re: Huntington's disease treated for first time

#39

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 laun…

> 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 frien…

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 , 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.

Re: Huntington's disease treated for first time

#40

More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…

It always seemed that an mRNA treatment was going to be the way forward for treating HD, speaking as an HD widower.

And here my government is actively working to suppress mRNA therapies because of fucking politics. Fuck them.

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