fda does not understand correctly yet and 80 percent of people are scientifically dumb.
body mass is the only factor and progression points.all the genes and exons in these boys are alike ,only different faulty exons .Age of progression is why different outcomes.
Look at the natural history of dmd and then look at these boys still walking.You have Physical evidence ,you have flat out stacks of real data for production of the missing element needed for slowing disease .Some think the human dystrohin gene is different in every boy ''it is not''.this gene has exons that are all the same in every boy, the only difference is the faulty exons ...So the truth is this ''if it works in just one boy it will work on them all.
IDRA IS ABOUT 8 YEARS BEHIND SAREPTA AND IDRA WOULD BE DOING MANY PATENT INFRINGMENTS BECAUSE SAREPTA OWNS THE MARKET ON 3RD GENERATION..FYI..
--------''SEE THE WORD'' PURSUE''
We are utilizing our GSO platform to pursue third generation antisense drug candidates for a variety of genetically derived disorders. Based on its validation in preclinical models, we believe our GSO technology has the potential to overcome the remaining hurdles of current antisense technologies, including efficient delivery without a carrier, reduced immunotoxicity, and increased potency
Sarepta's drug platform much better...
Isis Pharmaceuticals, Inc. (ISIS)-NasdaqGS Watchlist
59.55 2.07(3.60%) Dec 18, 4:00PM EST
|Pre-Market : 62.85 3.30 (5.54%) 7:17AM
31.00 to 33.00 ..under valued by far. I see a massive upside going into 2015 and beyond here.
check it out ,it is more to it.
It is kinda like what is going on with this company right now but will avail.True story, Sarepta did get the name from this town.
According to 1 Kings 17, the Prophet Elijah visited Sarepta and performed two miracles. It was a time of drought and famine, but the jar of meal and jug of oil in the widow's home continue to feed the widow, her son, and Elijah. Later, the prophet either cured her son of a serious illness or raised him from the dead (the text is not clear on this point). In remembrance of those events, Sarepta became a place of pilgrimage early on. In AD 382, the pilgrim Paula left "the ancient city of Sidon; on the shore of Sarepta, she entered the tower of Helias, in which she adored the Lord and Saviour ."(2) A century and a half later (AD 530), the pilgrim Theodosius recorded that there was a church of St. Elijah at Sarepta.(3)
Sarepta drug really worth with life time benefits for DMD .....wake up people..
Sarepta closer to a block buster drug approval and we are at 14.00...please note that sarepta's drug is the best in class and nearing approval for the first time in history.easy to see that selling was nothing more than tax loss selling for the short term investor.
What makes the most sense here is this.
biomarin bought prosensa just for the patents and only for a sarepta partnership in europe or for exon 51..biomarin could get monies back in one year of drug sales in the eu
few months ''tax loss for many for 2014 ''if you sell''
Recruiting Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: eteplirsen
2 Recruiting Phase I/II Study of SRP-4053 in DMD Patients Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Placebo; Drug: SRP-4053
3 Recruiting Confirmatory Study of Eteplirsen in DMD Patients Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: eteplirsen injection