How often, in drug development, do you think one wanders across a drug, that exhibits NO TOXICITY? I think this one single fact, is often overlooked, and under appreciated. I have a reasonable knowledge of a variety of different drugs, and I honestly can't think of one............not one............that , upon reading the PDR, or better yet, Drug Facts and Comparisons, has zero toxicity. I know Mainly, Jrrt, and others, are likely familiar with far more drugs than I am. Even so, drugs with no toxicity, are rare if I'm not mistaken, Very rare.
And yet, with one of the most devastating, intransigent diseases of our era, we have this new drug, that exhibits exactly that................ZERO TOXICITY. I find that incredible, meaningful, and sure, valuable - to the boys, parents, society, and of course, investors too. The fact that beyond this remarkable lack of toxicity, the boys are actually getting better........thriving if you will, blows my mind. The mapping of the human genome, suggested that someday we would be able to develop drugs like this, with remarkable effectiveness, yet minimal, if any, toxicity. And it seems to me, that SRPT gets to be one of the flag bearers as this paradigm shift in medicine design takes hold. I wonder if the day is near, when a young boy, exposed to Eteplirsen, or some improved (Dantrolene) version from early on in his life, is the first DMD boy to ever dunk a basketball.............. hit a home run... catch a touchdown pass........or better. Won't that be a hoot!
When things have looked gloomy, that has always been what I have used to keep the faith......and now with the boys still walking we can be confident it will happen. I share your wonder.....what will be the possiblities for boys that start receiving the drug before any symtoms are observed - especially since it seems to be absorbed during myogenesis.....Before the cells get leaky. Rats
Not to often or never. I was reading about SRPT on the yahoo group stockinvesting102 and many seem to be giddy about the prospects. I have read many posts on the groups board and I would like to understand if this is a pure spec play in which thats all there is or could this be a break through for many ill people. If this is the case, could you join the group there won't be as much filtering through the tons of posts to get to some real good info? As far as I have seen, the members on stockinvesting102 seem to know what their talking about when it comes to SRPT and other BIO plays, but i'm sure there is more info yet to hear about. Yahoo groups, then stockinvesting102, join group. Should someone just take a position and take the dunk or wait for another pull back?