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it helps some kids gain some strength, some who take the drug still die (49% in the initial trial showed no improvement)

It's perhaps the most impressive medication I've seen developed. Kids with SMA type 1 typically die in the first year of life, and they often never develop enough strength to even sit. There has never been any treatment, or anything close to a treatment, for SMA. Kids with SMA don't make SMN protein, which is needed for normal nerve function. Spinraza, through genetic manipulation, tricks the body into making SMN protein. Kids treated with Spinraza have sat and even walked when they otherwise would not have been expected to (the studies have only followed about 1 year of treatment). 40% of kids treated with Spinraza gained motor milestones, whereas 0% did in the placebo group. The results are incredibly impressive.
 
It's perhaps the most impressive medication I've seen developed. Kids with SMA type 1 typically die in the first year of life, and they often never develop enough strength to even sit. There has never been any treatment, or anything close to a treatment, for SMA. Kids with SMA don't make SMN protein, which is needed for normal nerve function. Spinraza, through genetic manipulation, tricks the body into making SMN protein. Kids treated with Spinraza have sat and even walked when they otherwise would not have been expected to (the studies have only followed about 1 year of treatment). 40% of kids treated with Spinraza gained motor milestones, whereas 0% did in the placebo group. The results are incredibly impressive.

so $750k is a fair price for society to bear for the first round of treatment, which will have a positive impact ~40% of the time?
 
It's perhaps the most impressive medication I've seen developed. Kids with SMA type 1 typically die in the first year of life, and they often never develop enough strength to even sit. There has never been any treatment, or anything close to a treatment, for SMA. Kids with SMA don't make SMN protein, which is needed for normal nerve function. Spinraza, through genetic manipulation, tricks the body into making SMN protein. Kids treated with Spinraza have sat and even walked when they otherwise would not have been expected to (the studies have only followed about 1 year of treatment). 40% of kids treated with Spinraza gained motor milestones, whereas 0% did in the placebo group. The results are incredibly impressive.

"We shouldn't be playing God, we've gone too far with science"
 
Pretty remarkable when you actually talk to the poors you work with about Obamacare about how much it matters to them. My staff insures her two kids for $28/mo. Our best 50 year old security guard disregards her employer's shitty insurance and gets obamacare for $200/mo. On the flip side it seems pretty much just fully subsidized healthcare, so just go all the way.

Stop taking on every SJW cause like trannys in bathrooms, get control of the government and throw up single payer already, dems.

The security guard better hope the IRS doesn't connect the dots regarding his usage of tax credits instead of enrolling in the crappy employer plan (assuming it meets ACA guidelines which at this point it likely does) or else they are going to be in a world of $$$ hurt.
 
The security guard better hope the IRS doesn't connect the dots regarding his usage of tax credits instead of enrolling in the crappy employer plan (assuming it meets ACA guidelines which at this point it likely does) or else they are going to be in a world of $$$ hurt.

I'm fairly sure the company is non-compliant, as I doubt it has 50 employees and it's paying min wages.
 
The security guard better hope the IRS doesn't connect the dots regarding his usage of tax credits instead of enrolling in the crappy employer plan (assuming it meets ACA guidelines which at this point it likely does) or else they are going to be in a world of $$$ hurt.

The security guard is a she, and she works extra hours at plama's firm cleaning and stuff to get that extra cash money take away.
 
so $750k is a fair price for society to bear for the first round of treatment, which will have a positive impact ~40% of the time?

I wasn't trying to defend the price with my post (it's too expensive). I was trying to give some info on the drug, which is an absolute game-changing medication. I thought the info might be interesting to some on here. It likely will have a positive impact in higher than 40%; one of the assessment tools in the study showed improvement in 63% of those treated (compared to 3% of placebo). It's possible it may have a positive impact in all patients treated with it, it just may take several years for that to occur. It takes a disease that is universally fatal, usually before age 1, and completely changes the prognosis. It is also possible that it may save a lot of healthcare dollars - kids may no longer need chronic ventilators, scoliosis surgery, recurrent hospitalizations for pneumonia, etc.
 
 
I wonder what percentage of Trumps "likes" and retweets are from the millions of bots that "follow" him.
 
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