Tofersen -Antisense for SOD1

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Tedstehr

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PALS
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08/2017
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CA
State
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New Westminster
I was at an ALS board meeting a few months ago and the top research scientist for ALS in BC spoke positively about an Antisense drug that showed improvement in SOD1 Familial ALS patients. I tried to research and I found Tofersen. Has anyone else heard more about this? I would have thought it would be big news.
 

Nikki J

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04/2014
Country
US
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It is in phase 3 and looking very hopeful. It is big news at clinic and in the FALS world. those involved in its development just received a prize at the MNDA symposium this month.

it seems to slow the most aggressive and most common form of sod1 but there have been a few anecdotal reports of improvement ( though at least some trial participants had to sign NDAs) and hints of same from doctors

the hope is given at or before onset it will stop it
 
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