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Deaf girl is cured in world first gene therapy trial

independent.co.uk

341–350 of 351 posts

Re: Deaf girl is cured in world first gene therapy trial

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post #337

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> Given that much of the point of the designation 'legally blind' is that the impairment is uncorrectable, this is a pretty unfortunate phrase imo, common as it is Why is this unfortunate? The designation also very much refers to the severity of the impairment, not just whether it's correctable. Perhaps this depends on the country?

Because the experience of someone whose impaired vision is fully corrected gives little to no special insight into what it is like to be blind. (If you spend years where the best vision you get is worse than, 20/200 or whatever, because you don't actually have access to adequate correction, that is actually relevant to discussions of blindness even if your vision is in principle correctable, sure.) Blind people have…

I misunderstood you - you were referring to GP's specific use of the phrase ("legally blind without my glasses") not just the phrase "legally blind"

As someone (actually) legally blind, I appreciate the reply :)

Re: Deaf girl is cured in world first gene therapy trial

#342
post #325

Earlier quoted context omitted.

FWIW, doctors usually specify brands over generics when it actually matters if you have the same supplier every time. Bioavailability of generics is +/- 20% compared to the original branded drug by law in the US. So if you have a legal, generic drug here, it might be a 120% one, and your next refill might be an 80% one - you just got a 1/3 reduction in dose without warning. And if you have seizures? That’s a bad thin…

> You can make a very good living as a physician in the US without being a whore. Not worth my moral sense to make a few more dollars that won’t go to me directly anyway. Certainly, but I was mostly referring here not to doctors who actually make more money due to pharma companies; but rather to doctors who are "lobbied" / "direct marketed" to by pharma companies, using steak dinners, free samples (implicitly, to dis…

I guess it works, but honestly this is standard operating procedure in every other business in the world. I can pay for a steak dinner myself; a “free” one that my wife can’t attend isn’t worth much to me. Samples, now, that’s getting to the heart of it. But, again, that’s how most businesses operate. Here’s a small freebie or a cheapie, if you like that, we can sell you a lot more.

Re: Deaf girl is cured in world first gene therapy trial

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post #324
post #305

Earlier quoted context omitted.

I think this is why the skyrocketing costs of healthcare in the US are so upsetting to me. Its sucking away the hope by putting so many of these technological miracles out of reach.

The high Healthcare costs in the US are caused mostly by high physician salaries though, and that's probably highly correlated with why they are able to make all these breakthroughs

Do you have a source for that? Physician salaries do not account for all that large of a portion of healthcare spending. A $300,000/year doc performs a lot of procedures annually, and the amortized amount isn't huge compared to other costs.

Re: Deaf girl is cured in world first gene therapy trial

#344
post #217

Earlier quoted context omitted.

What's the end goal of modern medicine though? Immortality? You can perhaps reduce suffering to nil, but you can't cheat death.

Why does there have to be an end goal? Why cant things just do the thing they do?

This attitude matches how my friends in medicine think. There's a thing causing suffering. Can we get better at preventing our treating it?

Re: Deaf girl is cured in world first gene therapy trial

#345

Earlier quoted context omitted.

Why can't you cheat death? Excluding accidents and the like.

Because it will utterly end all societal development. Imagine if fucking Ghengis Khan never had to die. Or Henry Kissinger. Or Carnegie Our system is struggling under the immense weight of old leaders and the best they can do is pump themselves full of weird drug cocktails to have some semblance of being alive. Imagine removing the last roadblock to eternal life for the richest human alive.

People would develop in different ways if they could live to be 1000. It would hardly end societal development. I imagine education would be a bigger deal, since you'd be in less of a hurry to get on with life at 18. Families would be larger as more generations were included, which could create more nurturing and supportive environments for at-risk children. You'd also have fewer trust fund babies living off of the inheritance from a dead relative. And the sheer amount of brainpower and life experience we lose every time a 90 year old dies is depressing; science, technology, and literature could all benefit from people living longer.

Re: Deaf girl is cured in world first gene therapy trial

#346

Earlier quoted context omitted.

It would be nice if people at these companies were rewarded the same way as food delivery app employees.

???? I would imagine the average surgeon is much happier with their compensation than the average DoorDasher. It's a reliable fixed-rate salary vs wondering if you'll get enough $3 tips to make rent this month.

Yes. Maybe this is an unpopular opinion, but I don't think doctors are overpaid. The amount of time and effort they invest in undergrad, sometimes additional college after that, medical school, residency, and sometimes a fellowship is more than I've ever been willing to put into my career.

Re: Deaf girl is cured in world first gene therapy trial

#347
post #325

Earlier quoted context omitted.

> You can make a very good living as a physician in the US without being a whore. Not worth my moral sense to make a few more dollars that won’t go to me directly anyway. Certainly, but I was mostly referring here not to doctors who actually make more money due to pharma companies; but rather to doctors who are "lobbied" / "direct marketed" to by pharma companies, using steak dinners, free samples (implicitly, to dis…

I guess it works, but honestly this is standard operating procedure in every other business in the world. I can pay for a steak dinner myself; a “free” one that my wife can’t attend isn’t worth much to me. Samples, now, that’s getting to the heart of it. But, again, that’s how most businesses operate. Here’s a small freebie or a cheapie, if you like that, we can sell you a lot more.

> But, again, that’s how most businesses operate. Here’s a small freebie or a cheapie, if you like that, we can sell you a lot more.

In this case, though, it's:

> We have this new drug, whose strict required dosing regimen and annoying, long-lasting guaranteed withdrawal effects mean that your patients will be very unlikely to stop taking it... ever... or even to switch medications once they start on it, without your repeated insistence. (They can exponentially taper the drug, but we will never provide instructions on how to do that, nor fund studies on the success rate of doing that. Your medical education will have taught you a linear tapering procedure, that does not work at all for avoiding this drug's withdrawal effects.)

> Here's some free samples that you can use to hook your poorest patients on our drug — you know, the ones you'd normally prescribe generics so that their not-very-good insurance plans will cover them, or so that they can afford them out-of-pocket. We'll be back with more pretty frequently, so if you don't have very many patients, you may just be able to treat some of your poorest patients indefinitely from free samples alone. (But we know this won't work out for you in the long term, as we've calculated what would be enough for a prescriber operating at patient capacity, and we shoot for maybe 1/3rd of that. It'll only seem like a good idea when you're just starting a new practice.)

> Oh, and we also do have an Assistance Program, because we know the insurance carriers refuse to pay for our drug (since it's not considered to be any more effective than the generics... just yet. Our lobbying hasn't fully kicked in yet.) Our Assistance Program will allow those same poorest patients to continue to pay for the drug out-of-pocket once you've hooked them with the free samples, rather than having them convert to the generic. They'll be paying at a painful — but reduced to just doable! — rate. We'll cover the rest. (And still make a profit!)

> It is, of course, a needs-based Assistance Program. We aren't in the business of slicing into our own (huge) profit margin for charity. We're in the business of helping people get over medical stumbling blocks that might be preventing them from being productive, and thus preventing them from getting a high-earning job. Our data says that our Assistance Program acts as an amazing self-funded channel partnership for us, in that it manufactures high earners pre-primed to buy our drug. And of course, we can bilk them for as much profit as we want.

This might not reflect your specialty — this is from my experiences in psychiatry.

Re: Deaf girl is cured in world first gene therapy trial

#348

Earlier quoted context omitted.

No, at least not without active electronics.

Fluorescence?

Going to reply to myself since I searched for it on a whim.. https://www.sumita-opt.co.jp/en/products/lumilass.html

Re: Deaf girl is cured in world first gene therapy trial

#349
post #341
post #340

Earlier quoted context omitted.

Because the experience of someone whose impaired vision is fully corrected gives little to no special insight into what it is like to be blind. (If you spend years where the best vision you get is worse than, 20/200 or whatever, because you don't actually have access to adequate correction, that is actually relevant to discussions of blindness even if your vision is in principle correctable, sure.) Blind people have…

I misunderstood you - you were referring to GP's specific use of the phrase ("legally blind without my glasses") not just the phrase "legally blind" As someone (actually) legally blind, I appreciate the reply :)

Yep! 'Legally blind' itself is often a pretty useful phrase, of course. :)

Re: Deaf girl is cured in world first gene therapy trial

#350
post #232

Earlier quoted context omitted.

>> so excited because future kids won't have to endure the same. When cochlear implants became routine there was a brief protest by the deaf and hard-of-hearing community. The line between people with a disability and a person who is simply different is a longstanding debate. How and when medicine should intervene is a hot button issue. I had a relative born with a malformed ear canal. The doctors rushed to get her t…

The deaf community is incredibly proud. I have a lot of respect for that. But at the same time, I don't understand it. I'm extremely visually impaired, and I've never seen the same "It's not a disability!" sentiment mirrored in the blind community. Does anyone have any insight why this might be?

Deaf ASL users have much less need to hear since their language doesn't require it. For that reason I suspect deaf folks generally find hearing less important than blind folks find seeing.

There are blind people like Daniel Kish who use echolocation to replace sight; I get the sense they're more on the difference side of the fence.

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