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Post by Corey on Dec 31, 2016 0:08:56 GMT -5
Holy hell! $125,000 per dose! Without knowing the costs to develop a drug with a small market, or knowing how many drugs never make it to market, we can't know what they need to charge. However it seems extreme This is true. But some background information A lot of the pre-clinical work on this drug was done by academic researchers... so cost was very low compared to other drugs. This pharmaceutical company is very aggressive with their patents which has prevented other researchers from working on similar drugs to treat SMA. Ones that may even work better than Spinraza.
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Holy Fuck
Dec 31, 2016 12:40:38 GMT -5
via mobile
Post by mikefatty on Dec 31, 2016 12:40:38 GMT -5
Holy hell! $125,000 per dose! Without knowing the costs to develop a drug with a small market, or knowing how many drugs never make it to market, we can't know what they need to charge. However it seems extreme This is true. But some background information A lot of the pre-clinical work on this drug was done by academic researchers... so cost was very low compared to other drugs. This pharmaceutical company is very aggressive with their patents which has prevented other researchers from working on similar drugs to treat SMA. Ones that may even work better than Spinraza. if that is true the FDA will surely be looking into it. also I agree with wonk. we really may never know how much they spent developing the treatment and there are other treatments for diseases and conditions that cost more for that reason. i think this is very interesting, mostly because I get an inside look from people with SMA I've already gleaned a lot from thus post.
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Post by wonk on Jan 1, 2017 3:43:55 GMT -5
So we all agree this will have no effect on adults with SMA? If it costs that much it better fucking work It reminds me of the thread "how far would you go for a cure" I am a bit of a selfish prick, so if a SCI "cure" that had even a 20% chance of being successful cost $5million, I would be out robbing banks and cooking meth tomorrow... actually that's a lie, it would be today!
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Post by newjess on Jan 2, 2017 15:20:04 GMT -5
Just trying to clarify... So darthoso mentioned 15 was the oldest age to get into clinical trials. Is there any literature that is claiming (or implying) that this could help adults with SMA? Or is it kind of being extrapolated from its usefulness to kids?
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Post by darthoso on Jan 2, 2017 15:34:27 GMT -5
Just trying to clarify... So darthoso mentioned 15 was the oldest age to get into clinical trials. Is there any literature that is claiming (or implying) that this could help adults with SMA? Or is it kind of being extrapolated from its usefulness to kids? The study that included the older kids was to evaluate safety. Since it seems safe in both and useful in kids, they didn't feel a need to delay it further.
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Holy Fuck
Jan 2, 2017 15:46:47 GMT -5
via mobile
Post by newjess on Jan 2, 2017 15:46:47 GMT -5
Just trying to clarify... So darthoso mentioned 15 was the oldest age to get into clinical trials. Is there any literature that is claiming (or implying) that this could help adults with SMA? Or is it kind of being extrapolated from its usefulness to kids? The study that included the older kids was to evaluate safety. Since it seems safe in both and useful in kids, they didn't feel a need to delay it further. Ah.. I gotcha. So here's another question. SOR and I were debating the similarities and differences between SMA and MD. How are they related to each other? And, could a treatment like this help someone with MD, or is thar yet to be seen (or, maybe it has nothing to do with MD)?
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Post by Corey on Jan 2, 2017 16:04:35 GMT -5
The study that included the older kids was to evaluate safety. Since it seems safe in both and useful in kids, they didn't feel a need to delay it further. Ah.. I gotcha. So here's another question. SOR and I were debating the similarities and differences between SMA and MD. How are they related to each other? And, could a treatment like this help someone with MD, or is that yet to be seen (or, maybe it has nothing to do with MD)? The difference between SMA and MD is that SMA is more a nerve disease, specifically the nerves that go to muscles. People with SMA have normal muscles for the most part, they just lose the ability to use them, whereas in MD it's the muscles cells that are usually affected in some way. This exact treatment would not help someone with MD, but a similar treatment would work and there are some in development. This treatment is specifically designed to correct a certain defect in one particular protein, defective in SMA. So its a very targeted approach. Some cases of DMD or BMD have a similar defect, just in a different protein. So you would just have to design a new drug for that defect/protein. Its really not too complicated and I think these new class of drugs will become popular in the future. If youre curious, look up anti-sense oligonucleotides, which is the type of drug this is. Its RNA, so very different from traditional pharmaceuticals. People are just extrapolating if they think it would work in adults but no one really knows. There are some studies in SMA mice where they receive this treatment or one similar to it. If you gave the treatment late, such as after symptoms showed up, it generally had no effect. I can think of some reasons why it might work better in humans but it's hard to say.
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Post by darthoso on Jan 2, 2017 16:33:31 GMT -5
The study that included the older kids was to evaluate safety. Since it seems safe in both and useful in kids, they didn't feel a need to delay it further. Ah.. I gotcha. So here's another question. SOR and I were debating the similarities and differences between SMA and MD. How are they related to each other? And, could a treatment like this help someone with MD, or is thar yet to be seen (or, maybe it has nothing to do with MD)? I'll give you and SOR my opinion on effectiveness in exchange for lots of shots when I get the needle at Duke.
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Post by Emma on Jan 3, 2017 1:27:12 GMT -5
So you are doing it for sure?
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Holy Fuck
Jan 3, 2017 1:56:33 GMT -5
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Post by newjess on Jan 3, 2017 1:56:33 GMT -5
I was going to ask the same thing as Emma, are you going to do it?
Definitely keep us posted!!
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Holy Fuck
Jan 3, 2017 8:53:30 GMT -5
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Post by darthoso on Jan 3, 2017 8:53:30 GMT -5
I haven't been to an MDA Clinic in ten years (oops), so I'm gonna go to the one at Duke in a few weeks once I get an appointment and we'll discuss it.
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Post by Inigo Montoya on Jan 3, 2017 9:06:23 GMT -5
I haven't been to an MDA Clinic in ten years (oops), so I'm gonna go to the one at Duke in a few weeks once I get an appointment and we'll discuss it. They should play this as you enter the building. Lol
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Post by Mets on Jan 3, 2017 13:20:34 GMT -5
Gonna watch the informational webinar at 2, should hopefully answer some of people's questions
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Post by darthoso on Jan 3, 2017 15:49:11 GMT -5
That was high quality horse shit.
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Q
Full Member
“Follow your bliss." Joseph Campbell
Posts: 157
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Post by Q on Jan 6, 2017 19:41:05 GMT -5
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