Jun. 16 at 1:33 AM
$SRPT it's also pretty clear to me at this stage that most bulls here bought after the 1st death, thinking it's some kind of generational discount. I bring the bad news. And I was mocked before for making these obvious, peer-reviewed statements, that are scientifically true.
1. Microdystrophin does not behave like normal dystrophin. They are way too short. No amount of uDystrophin is good enough.
2. Duchenne is a muscle stem cell division disease. Dumont et al. 2015 has proved this. If you don't fix that part, you can't help Duchenne patients.
3. A successful Duchenne drug must therefore either deliver dystrophin into the stem cells (
$DTIL's PBGENE-DMD) or fix stem cell polarity (
$MSCLF's SAT-3247). One has shown steady strength increase in mouse, the other doubled grip strength in adult patients.
So good luck to all, but remember, biotech is as much luck as it is about understanding mechanism of action and science.
