Bojian Refuses Sympathetic Use of Experimental ALS Tofersen Drug

[Richard]

Their decision is like putting a stumbling block before the blind.

 

[Jean-Pierre Le Rouzic]

I blogged about it on padiracinnovation.org but Tofersen is not a drug targeting ALS, it is targeting a subset of people having SOD1 ALS. So the people who may benefit from it are less than 2% of ALS cases.
It’s already extremely difficult to find enough pALS that meet all requirements, finding people meeting rare conditions makes conducting clinical trials even more complex. I suppose that if Biogen provides Tofersen under compassionate use, they could stop their clinical trial today as pALS who progress will quit the trial. No one will gain if this happens.

[Richard]

Thanks for the info.  No simple solutions.