FDA Orphan drug status of CUatsm

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Samkl

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Can anyone tell me what the granting of this status for copper atsm means for patients in the US?
 

lgelb

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This is the record for the current trial, which is scheduled to conclude at year's end.

Orphan drug status provides financial incentives for developing drugs that benefit patients in certain diseases such as ALS. It does not directly speed up availability.

Other special FDA programs, such as fast track, priority review, rolling review, and accelerated approval do speed up availability on the market somewhat (generally reducing the wait by months, not years). Most anything that looks effective in ALS will qualify for at least some of these. However, in any scenario, given how long data analysis and even rolling application submission takes, we couldn't anticipate an approval before 2022.
 
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Samkl

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My brother is 12 days in on this trial, and in reading about it, I came across the info about the orphan drug status. Btw, when you hear of “orphan” conditions, I thought it meant rare. I hardly tell anyone about my brother’s diagnosis without them knowing someone, even if not directly, who has, or had ALS. I seem to hear of more people with ALS than Parkinson’s Disease, for example. My impression is it’s a lot more common than I thought.
 

Nikki J

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ALS happens about as often as MS. It seems rarer because PALS die much more quickly.
 

jethro

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I thought it was a typographical error. If eyes lied to me, ears didnt. I heard him say!
Can't believe this (0:14):
comments?

R. Bedlack: "The disease ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig's Disease, affects one in 600 people. There currently is no cure. The Duke ALS Clinic is one of the largest ALS clinics in the world, with one of the most comprehensive multi-disciplinary teams in the world. Philanthropic support plays a key role in helping Duke researchers accelerate ALS breakthroughs, and reach the ultimate goal - a cure.
If it was not Bedlack, i would jump over it. Or I am lost in translation since English is not my native l.
There are app 2500000 people with MS. Noone knows how many pals are in the world. No registry... according to this statement, there about 12500000 (5xmore than ms), source: Multiple sclerosis - Wikipedia.
 
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EricInLA

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The number I’ve always heard is 1 in 1000 will get it over the course of a lifetime. Nikki is correct regarding the distortion in the numbers. Anyway i love Bedlack’s optimism - I really want to believe what he says.
 
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