4 ms·

you can't just simply state that he's healthy without actually seeing his lifespan

by arpanetus 4y ago

sobeston 4y ago
You claim to be healthy, yet you have not died yet. Curious.
- mike741 4y ago
  where did they claim to be healthy?
smt88 4y ago
You can be healthy now and developer cancer next year. They're looking at whether this child shows signs of illness, and he doesn't. That's the same definition of healthy that we use for everyone else.
perlgeek 4y ago
The headline claims the child is healthy, but the article itself says > Roughly six months out from treatment, "Teddi is a happy and healthy toddler showing no signs of the devastating disease she was born with," the NHS statement reads. and later > In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms; these patients were able to break down sulfatides at normal rates and showed typical patterns of motor development, for example. The benefit of the therapy seemed to last several years, but at this point, "it is not yet clear whether it will persist life-long, and extended follow-up is needed," the EMA noted. Which is much more nuanced.
refurb 4y ago
You cant but you can place a very high probability on it. With genetic diseases often the difference between healthy and not is a single enzyme is missing. If you can prove the enzyme level is restored and all the downstream effects match healthy individuals, youve basically restored them to what healthy people are. Durability of the change is the big issue. Some gene therapies wane over time. And of course if damage had occurred before the gene therapy, that may never go away.
neogodless 4y ago
Your assertion is that we have no way of assessing someone's health until they have died?
- arpanetus 4y ago
  My assertion is that the every change we do towards things like that might involve million results and since it's not a simple logical statement it's rather hard to verify/validate the outcome in a short term due to the nature of dynamic/complex systems.
ChrisKnott 4y ago
You could have read the article to the third word at least, to learn that the child is a girl.
- arpanetus 4y ago
  my bad, I didn't read the article, just followed the title

you can't just simply state that he's healthy without actually seeing his lifespan

sobeston 4y ago

You claim to be healthy, yet you have not died yet. Curious.

mike741 4y ago
where did they claim to be healthy?

smt88 4y ago

You can be healthy now and developer cancer next year. They're looking at whether this child shows signs of illness, and he doesn't. That's the same definition of healthy that we use for everyone else.

perlgeek 4y ago

The headline claims the child is healthy, but the article itself says > Roughly six months out from treatment, "Teddi is a happy and healthy toddler showing no signs of the devastating disease she was born with," the NHS statement reads. and later > In clinical trials, Libmeldy offered clear benefits to infantile and juvenile patients who hadn't yet developed MLD symptoms; these patients were able to break down sulfatides at normal rates and showed typical patterns of motor development, for example. The benefit of the therapy seemed to last several years, but at this point, "it is not yet clear whether it will persist life-long, and extended follow-up is needed," the EMA noted. Which is much more nuanced.

refurb 4y ago

You cant but you can place a very high probability on it. With genetic diseases often the difference between healthy and not is a single enzyme is missing. If you can prove the enzyme level is restored and all the downstream effects match healthy individuals, youve basically restored them to what healthy people are. Durability of the change is the big issue. Some gene therapies wane over time. And of course if damage had occurred before the gene therapy, that may never go away.

neogodless 4y ago

Your assertion is that we have no way of assessing someone's health until they have died?

arpanetus 4y ago
My assertion is that the every change we do towards things like that might involve million results and since it's not a simple logical statement it's rather hard to verify/validate the outcome in a short term due to the nature of dynamic/complex systems.

ChrisKnott 4y ago

You could have read the article to the third word at least, to learn that the child is a girl.

arpanetus 4y ago
my bad, I didn't read the article, just followed the title