There are now videos of Nolan and Jack Willis posted by the Muscular Dystrophy Association on Youtube. These are the famous "non-ambulant" twins of Sarepta's trial, and, WOW, do they look great! They're swimming around, looking healthy and happy, and looking forward to a long life that includes college!
So this is it: the last lingering doubt about eteplirsen has been answered. The video is well worth a watch. A feel good moment for investors and non-investors alike.
"The research is outstanding and unprecedented" - just wait until some idiot says s.th. not right with their dystrophin. Or says Sarepta is only hyping ho-hum data from a small study instead of running a really good and large trial. Pathetic.
I do not think FDA is corrupt or stupid. Painstaking for sure. As soon as Sarepta has drug product ready that meets quality requirements and FDA is satisfied with the process they will approve, IMO.
It would truly be amazing if these kids could live a normal life. Maybe more like a Becker's MD life, but a life. Earlier treatment is the key, but maintaining upper body strength and cardiac/lung function is the real focus.