Diagnosed with familial ALS

MadCybertist

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Lost a loved one
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I was diagnosed with familial ALS secondary to SOD1 mutation. I begin Riluzole tomorrow and the ALS clinic is in the process of trying to get me into the Toferen clinical trial.

I’ve spent some time getting my affairs in order, but the one remaining item is our house. It’s 2 stories and I would really like to find a good way to prep it for wheelchair access. That’s probably my last hurdle I have left to work on. Any suggestions are welcome! Thanks.
 

Nikki J

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PALS
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Sorry and not sorry. If you have to have ALS sod1 is the kind to have. I don’t think you mean clinical trial though. The only active tofersen trial is ATLAS for carriers. There is an eap for PALS. If your clinic is willing to cooperate the biogen part is usually not a problem with a diagnosis and a documented sod1 mutation google Chris Snow to read how early tofersen has helped him also which variant? Chris has an aggressive one. People with less aggressive variants have said the have been stable for several years.

if you are staying in the house do you have a bath and space for a bed if not a bedroom on the first floor? Generally a ramp to inside, threshold ramps and decluttering
 

MadCybertist

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Lost a loved one
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I don’t have the number offhand but it was with Biogen and found on clinicaltrials.gov. That’s why I said clinical trial. Is Tofersen readily available otherwise?

We have 2 rooms downstairs we can make a bedroom. I don’t have a shower though on the first floor (3 upstairs haha). I do have a mud room and laundry room though. The laundry room has a laundry tub in it so I’m thinking about getting stacked washer/dryer to make more room and then taking the tub out and installing an accessible shower. We already have a 1/2 bath with toilet and sink as well.
 

Nikki J

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PALS
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It sounds like your house is workable which is fortunate.

as I stated there is not a current trial for tofersen for PALS. The phase 3 finished some time last year. There is an eap which is the only way newly diagnosed pals can access it ( people who were in the trial continue to get it through open label extension). I am 100 % sure this is fact. The ATLAS carrier trial is the only active tofersen trial. are you referring to this? It is an eap Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis - Full Text View - ClinicalTrials.gov . As long as you can get it eap is better than a trial no placebo

it is definitely worth knowing and learning about your variant as different ones have different progression
 
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