Nikki J
Moderator
- Joined
- Mar 22, 2012
- Messages
- 16,380
- Reason
- PALS
- Diagnosis
- 04/2014
- Country
- US
- State
- MA
- City
- Boston
They will monitor neurofilament monthly. When it rises ( which in SOD1 is a sign of imminent symptom onset) they will be randomized into tofersen ( the sod1 aso currently in final trial phase) and placebo. Anyone who then develops ALS will go into an open label extension. Of course the hope is that onset will be prevented or at least delayed.
ClinicalTrials.gov
www.clinicaltrials.gov