While the drug is promising, there are still substantial risks:
1. sample size of 4 2. we know the drug didn't work for the 2 kids who lost ambulation during the trial and whose results were not included. Maybe it will work for them in time. 3. Early approval is a coin flip- most likely the FDA will require a phase III that will be be expensive. 4. We don't know how the body will tolerate the new dystrophin over time 5. There are competing drugs and approaches to treating DMD. Competition still exists- for example, a cure for the disease would wipe out the need for eteplirsen.
At any rate, I remain long and hopeful that the drug works. But the company needs to raise some cash now to give them some breathing room. Just my opinion.