From the Oct. 3 article entitled, "DMD: Eteplirsen Results Very Encouraging at 48 Weeks" (viewed from mda.org), it is stated halfway down the article, "Eteplirsen is designed to skip exon 51 of the dystrophin gene, and potentially could benefit about 13 percent of boys with DMD." If 1 in 3600 boys per year worldwide are affected by DMD, then this drug could only potentially benefit 468 of those boys. I, maybe like many others, bought in way too soon with a 30 average and I don't expect to get my money back. I am just hoping for a lesser loss sometime in the future. Good luck to you if you invest in this - a massive risk it is, no doubt.
It will work on all 87% of other exons in Duchenne, and should work on other MDs like ALS, SMA, just to name a couple. This exon skipping platform has unlimited potential, as well the anti viral and anti bacterial platforms.
This company has the potential to revolutionize medicine as we know it. That is why $45,or $7 and change, pre reverse split is still a rediculously low pps. The shorts who are playing this like other biotechs will eventually be crushed. It's not over until the fat lady sings... JMHO