recently diagnosed C9

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azmully

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Lost a loved one
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11/2019
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Hi everyone,

I was here on the forum for questions about my larger family's history with ALS (c9) and my own personal situation back in June/July of this year. I shared my EMG on this forum back then and was waiting to be seen by the Barrow Neurologist here in Phoenix, AZ.

In August I was diagnosed with ALS by my neurologist based on my limb onset, clinical weakness and EMG. A few weeks later the DNA test confirmed the C9 gene.
Fast forward to today and it's been a whirlwind of emotion and getting things arranged. I live just 2 miles from Barrow and they are part of the Healey ALS Platform trial and I just started this Monday. I was randomly assigned zilucoplan and have 1:3 of change of getting the drug Vs. the placebo. This is only 24 weeks so a short trial, ideally I think a C9 focused trial would be best but this is very close and easy for me and I'm very happy with the team.

Has anyone else taken zilucoplan? Just started the injections this week.

Thanks,
Mike
 

KimT

Extremely helpful member
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Nov 18, 2014
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4,079
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08/2015
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Hi Mike,
Sorry to welcome you. I'm not FALS but Nikki will have lots of helpful information on possibilities.
We're here for you.
 

azmully

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Joined
Jun 29, 2020
Messages
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Reason
Lost a loved one
Diagnosis
11/2019
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US
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AZ
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Phoenix
Thanks Kim, and yes Nikki has been a really great resource for my past questions.
 

Nikki J

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9,847
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Hi Azmully

I was thinking of you the other day and wondering what happened. Sorry that you ended up diagnosed

the platform trial has promising therapies and I hope you have success.

there are several ( maybe 5-6) c9 specific trials expected it 2021. Maybe the timing will work that you can finish the platform, wash out and morph into one of them. I don’t know how many centers will have the new ones though

we are hoping that the biogen aso phase which is finishing up cohort 5 ( currently the last phase 1 cohort) will report out good data and move to a pivotal trial as sod1 tofersen did. The other possibilities are a cohort 6 being added to phase 1 at a higher dose or failure and back to the drawing board. The doctors I have spoken to are optimistic it will not be the third option

another aso ( wave life sciences) is expected to start phase 1 and the other trials are targeting known c9 pathologies.

if you have not already done so you might ask the trial coordinator if you are allowed to take Metformin. I have not seen it on the list of exclusions but suspect you will not be able to have it. Its proposed mechanism of action is c9 specific so may be that is why. Metformin a diabetes medicine is in trial in Florida for c9 after being shown in the lab to lower RAN protein found in C9. It is worth asking about.
 

azmully

Member
Joined
Jun 29, 2020
Messages
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Reason
Lost a loved one
Diagnosis
11/2019
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AZ
City
Phoenix
Thanks Nikki! It's been a good week I was just fitted with leg braces so I can walk much better and was able to take my Brittany out today for a short jaunt. I think my progression is slow since my initial symptoms were 8-9 months before my first failure ( foot / leg ) in June and my diagnosis in late August but who know's for sure on progression since my right leg is starting to weaken now.

I really hope to roll into a C9 study once this 24 week trial ends. My neurologist concurs that a C9 study would be best.

My trial coordinator did list a ton of exclusionary drugs/supplements but I hope Metformin is not on that list.

THANK YOU for the lead on this!
 

BarrCarr

New member
Joined
Feb 23, 2020
Messages
6
Reason
PALS
Diagnosis
05/2019
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FL
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Valrico
We are in the same club we do not want to be in - C9. Reach out if you want to compare notes. first symptom 3/17.
 
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