Huntington's disease treated for first time

Post by meindnoch » Wed, Sep 24, 2025, 9:28 PM UTC

w10-1 wrote: post #69

(non-expert) This is promising but needs publication and expert review. Here's the actual company statement from today: https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-positive-topline-results-pivotal-phase-iii There's also a June 2024 article: https://www.cgtlive.com/view/huntington-disease-gene-therapy-nets-rmat-designation That explains a bit more: (1) neuro-surgery introduces gene…

Post by software-is-art » Wed, Sep 24, 2025, 9:59 PM UTC

senkora wrote: post #81

kennywinker wrote: post #56

Earlier quoted context omitted.

While technically possible, that option isn’t really available to most people. Tho I suppose 17 hour brain surgery with gene therapy is also not available to most people, so fair enough. Anyway, they’re just describing the heritability of the disease there.

An optimal society would provide no-cost embryo screening and IVF to couples with the gene for HD, because that cost is certainly far I hope that we can work towards such a society. (And of course this research is still worth doing to help the population who already have the illness)

Post by eszed » Wed, Sep 24, 2025, 10:10 PM UTC

mjburgess wrote: post #32

ksajadi wrote: 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 deniali…

Post by kens » Thu, Sep 25, 2025, 12:02 AM UTC

kulahan wrote: post #64

sleight42 wrote: post #37

Earlier quoted context omitted.

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.

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.

Post by derektank » Thu, Sep 25, 2025, 1:36 AM UTC

conception wrote: post #53

drstewart wrote: post #44

Earlier quoted context omitted.

[flagged]

You missed the second part of that sentence buddy. Your examples would match if every base commander had to write public messaging on their bases’ budgets and projects.