Interesting. Thanks for the info. But, at over $3B market cap, that puts ISIS well into the mid-cap range for me. Still need that small-cap (<$500M market cap). ; )
It's completely unclear right now (at least to me, and this is an area I follow pretty closely) what that would actually mean.
The most promising ALS work I know of is the recent publication out of Columbia showing that astrocytes from ALS patients produce an as-yet unidentified toxin that kills motor neurons via the necroptosis pathway.
(Necroptosis is a fairly recently discovered alternative death pathway to apoptosis).