the FDA slow movement involved..........
these crack pots .....you got to say to yourself is this legal..haaaa
you guys have at it for now but really your days are numbered..
what a scam to get to short a stock........................the day is coming for you boys
did you see vertex ''vrtx'' what it did today and this is a far better drug than what they have!
The smart move for the fda is to approve this drug on the 120 week or the 144 week data but run the confirming trial anyway,
you're just a short or a short term holder that cannot aford to wait on a stock and wants to blame everyone for your choices ..advice :never invest any monies if you are afraid of losing.
I do not tell big stories for greed ,i research and invest real long term and the swings don't bother me one bit.Will not bother me short term .i will just add if i can get in cheaper.
You see a circle of certain law firms pumping in the same stories and certain articals popping up to imply negative thoughts these people are more than likely on the same team.
getting played by the market place shorts.This happens when news is dry,Sarepta is a fine Company and will show itself in time.Just big market crooks controlling short term monies.
Dystrophin production was seen in all patients in a time- and dose-dependent manner, with 47 percent of muscle fibers producing dystrophin after 48 weeks, with a dystrophin intensity of about 15 percent of normal. Dystrophin production was complemented by an increase in components of the dystrophin-glycoprotein complex, including sarcoglycan and neuronal nitric oxide synthase, proteins that are essential for normal muscle function.
Natural history studies have shown a progressive decline in the 6MWT distance, with a loss of roughly 1 meter per week after age seven. Boys treated from baseline with eteplirsen declined a mean of 16 meters during the first 36 weeks, and remained essentially stable for the next 84 weeks. Those initially on placebo declined by about 70 meters during the first 36 weeks, “and then reached a stable walk distance for the remainder of the study,” Dr. Mendell said.
Maximum inspiratory pressure and maximum expiratory pressure, highly sensitive measures of pulmonary function, decline annually by an average of 3.9 percent and 3.6 percent, respectively in DMD. In contrast, there was “remarkable stability” in these measures over the entire course of the trial, Dr. Mendell said, which represents a significant departure from the expected decline in Duchenne muscular dystrophy natural history.”
The drug was very well tolerated, with no differences in treatment-related adverse events between groups, except for slight elevations in urine protein in 5 samples, out of more than 100 taken.
source of story telling shorts..
19 June 2014 - Volume 14 - Issue 12 - p 24-25
NEWS FROM THE AAN ANNUAL MEETING: Exon Skipping for Duchenne Muscular Dystrophy Moves Closer to the Clinic
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ARTICLE IN BRIEF
Investigators reported new data on two exon-skipping therapies for Duchenne muscular dystrophy (DMD), eteplirsen and drisapersen, which independent experts say is good news for the DMD community.
PHILADELPHIA—In what one expert is calling “the best news the Duchenne muscular dystrophy [DMD] field has ever had,” long-term results of exon-skipping therapy with eteplirsen indicate its potential to stabilize walking ability of boys with DMD over the course of 120 weeks of treatment. While the trial was in a small number of patients and the drug was given open-label for most of the time, the results are nonetheless encouraging enough that the Food and Drug Administration (FDA) has laid out a pathway for approval of eteplirsen, and its manufacturer is gearing up to provide enough drug for clinical use as early as 2015.