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First UK child to receive gene therapy for fatal genetic disorder is now healthy

livescience.com

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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#121

Post by CommanderData »

nradov wrote: post #48
CommanderData wrote: post #41

Earlier quoted context omitted.

Don't some insurers outright deny cover for chronic or genetic conditions?

In the US, medical insurers are not allowed to deny coverage for chronic or genetic conditions due to the Genetic information Nondiscrimination Act of 2008 (GINA) and the Affordable Care Act of 2010 (Obamacare). Some treatments may require proof of medical necessity, or require that providers and patients try lower cost options first (step therapy). Rules for life insurers are different and in some circumstances they…

Thank you. In the UK I've been told our private insurers don't cover chronic conditions in most work group policies.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#125

Post by Nasrudith »

moremetadata wrote: post #112

Thats a new stealth weapon for the global population to watch out for now, knowing how things work in this country.

Said "stealth weapon" has been running wild for millennia. Our DNA has long been messed with by the environment, well before we knew what DNA or a virus were. The difference is now it is possible to have DNA altering viruses which aren't "almost certainly guaranteed to only do bad things to you".

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#126

Post by chrisgd »

mmaunder wrote: post #61

I wonder if a similar approach can be used for Von Hippel Landau. “ Mutations in the VHL gene cause von Hippel-Lindau syndrome. The VHL gene is a tumor suppressor gene, which means it keeps cells from growing and dividing too rapidly or in an uncontrolled way. Mutations in this gene prevent production of the VHL protein or lead to the production of an abnormal version of the protein. An altered or missing VHL protein…

I think CAR-T and cell / gene therapy is going to cure a lot of these orphan diseases with enough time. They really seem like miracles. Carl June winning a Nobel needs to be a bet on DraftKings. We have seen 10 years of remission with some ALLs in children

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#127

Post by toast0 »

TulliusCicero wrote: post #83

> Teddi and her sister Nala, age 3, were both diagnosed with MLD in April 2022, according to the NHS. Unfortunately, Nala was not eligible for Libmeldy because she'd already developed symptoms of the disorder. Holy shit. My god, I'm just thinking of the parents. "Sorry, we can only save one of your kids." "You can't give Nala the treatment?" "Well, we could . We won't, though." I'm sure there are good reasons, but Je…

> In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms

Does that mean clincal trials didn't show a benefit for patients who had already developed symptoms? Does it mean it wasn't studied in patients who had already developed symptoms?

I don't think it's unreasonable that the NHS won't spend large amounts of other people's money on a treatment that hasn't been studied or wasn't found to be effective.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#128

Post by refurb »

jiggawatts wrote: post #5

https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .

Of course, why are you surprised?

You have X amount of dollars. You can spend Y percent on healthcare. How do you spend Y to have the greatest benefit? Save 1 patients with a $300,000 gene therapy, or save 1,000 patients with effective treatment for their diabetes.

Until we have unlimited resources, that trade off will always be made.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#129

Post by refurb »

elil17 wrote: post #46
sn0wf1re wrote: post #38

Earlier quoted context omitted.

What sort of regulation? Make it cheaper by x% per year or we stop using it? Doesn't sound that dissimilar to "make it cheaper than the total cost of the current treatment or we won't use it".

I’d just change the “or we won’t use it” to “or your patents go away.”

Sounds like a great way for people to just not invest in new therapies.

"Hey, that's a nice life-saving treatment you spent $500M developing. It would be a shame if we just took it from you."

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#130

Post by refurb »

shadowgovt wrote: post #28
hummus_bae wrote: post #14

Earlier quoted context omitted.

Another country where nationalized healthcare is a bad idea.

Not sure I follow. It's not like a therapy with a sticker price of $3.8 million is more available in countries without nationalized healthcare.

Sure it is.

Look at the Blue Cross Blue Shield plan for Michigan. It covers a number of gene therapies at a cost of $2.2M for one of them.

Not all European countries pay for it.

https://www.bcbsm.com/amslibs/content/dam/public/employers/d...

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