I remember reading a bit anout them some years ago. And think I...

I remember reading a bit anout them some years ago. And think I posted something on an ANP thread.

The screening for the their phase 3 trial was very limiting. The end points they selected were also open to some questioning, with the stair climb finally being decided upon.

Have a look at how this drug went in an adult trial for another indication (cant remember what it was, will research and get back to you). The adult trial, with same drug, was abandoned, as no evidence of restoring muscle or any improvement was found. This trial drove the screening for the DMD trual. That being they wanted little to no degeneration evident before dosing.

The MRI evidence they have is a good sign for them. But target market will be very small.