ALS C9orf72

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GXTrex

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Loved one DX
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I think know you need to be confirmed positive now to save money to use on c9 positive individuals. If you were taken in before this policy was enacted, I assume you would be grandfathered in.

I asked about age because Dr Harms told me he expects pre symptomatic treatments within the next 10 years. Nothing is guaranteed of course but is good to hear.
 

Nikki J

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I hope presymptomatic c9 treatment will at least be in trial a lot sooner than 10 years. Biogen is getting ready to start a presymptomatic trial for tofersen the sod1 aso. The c9 aso started its trial about 2 2/3 years after tofersen started. IF it helps symptomatically they want to trial it for c9 as soon as possible. There is an issue that neurofilament which is the biomarker the tofersen trial is using may not be as good for c9. A researcher told me she thought c9 might need a biomarker panel. The only way to get these are carrier studies. No one wants to have a possible treatment but no biomarker
 

GXTrex

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Yes Nikki I agree hopefully sooner. I believe he was being cautious with me. He said once thy find the biomarker that indicates the onset of c9 als, those with the gene would get their levels tested yearly. Once the biomarker reaches a specific level treatment would begin.
 

AniSk

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Feb 20, 2021
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Lost a loved one
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I am going for my appointment on 3/17 so I can share any new info I get once that is complete. Yes, I assume I’m grandfathered in. When I went for the initial visit 2 years ago, it was in the consent that nobody involved would know the status of the genetic testing, unless I decided at some point to find out. I believe that I was supposed to go yearly but last year never happened due to covid. I definitely hope that there will be new developments for pre-symptomatic carriers soon. My dad was 70/71 when his symptoms began, my aunt was 67/68 and his dad was around 70, so hopefully I do have some time that, if I am a carrier, a preventative can be developed. I would probably want to find out my status and participate in a trial once they begin.
 
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