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.
Decline......these boys in the Sarepta trial should be in wheelchairs by now ,a minor decline if any would be 1000 times better than full decline.
very correct.....it is a point of the progression where it is to late as the dystrophin as a endpoint , it is only relevant to the'' not to of older time frame'' of the disease .after that i would believe the drug would only be a compassional use drug.''keeping the dmd young man from a fast death ''
Publication number US20140024821 A1
Application number US 13/708,708
Publication date Jan 23, 2014
Filing date Dec 7, 2012
Priority date Dec 8, 2011
Also published as US20140024698, WO2013086441A2, WO2013086441A3, WO2013086444A2, WO2013086444A3, Less «4 More »
Publication number 13708708, 708708, US 2014/0024821 A1, US 2014/024821 A1, US 20140024821 A1, US 20140024821A1, US 2014024821 A1, US 2014024821A1, US-A1-20140024821, US-A1-2014024821, US2014/0024821A1, US2014/024821A1, US20140024821 A1, US20140024821A1, US2014024821 A1, US2014024821A1
Inventors Ryszard Kole, Richard Keith Bestwick
Original Assignee Sarepta Therapeutics, Inc.
Export Citation BiBTeX, EndNote, RefMan
External Links: USPTO, USPTO Assignment, Espacenet
The whole point was this..
repairing severe damage from the point of no return....''would be like trying to give a cancer cure to someone that is getting ready to pass away.
keep in mind that it would take 189 days or around that to just produce Dystrohin so it is my opinion that this should go beyond 1 year for older human subjects before a concustion is to be made .I myself think this is a waisted study without a propper time frame even if it is mice .]]mice should be tested for 6-10 times the time frame in those groups
Gene Therapy , (19 June 2014) | doi:10.1038/gt.2014.53
Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice
B Wu, C Cloer, P Lu, S Milazi, M Shaban, S N Shah, L Marston-Poe, H M Moulton and Q L Lu
AbstractAntisense therapy with both chemistries of phosphorodiamidate morpholino oligomers (PMOs) and 2′-O-methyl phosphorothioate has demonstrated the capability to induce dystrophin expression in Duchenne muscular dystrophy (DMD) patients in phase II-III clinical trials with benefit in muscle functions. However, potential of the therapy for DMD at different stages of the disease progression is not understood. In this study, we examined the effect of peptide-conjugated PMO (PPMO)-mediated exon skipping on disease progression of utrophin-dystrophin-deficient mice (dko) of four age groups (21–29, 30–39, 40–49 and 50+ days), representing diseases from early stage to advanced stage with severe kyphosis. Biweekly intravenous (i.v.) administration of the PPMO restored the dystrophin expression in nearly 100% skeletal muscle fibers in all age groups. This was associated with the restoration of dystrophin-associated proteins including functional glycosylated dystroglycan and neuronal nitric synthase. However, therapeutic outcomes clearly depended on severity of the disease at the time the treatment started. The PPMO treatment alleviated the disease pathology and significantly prolonged the life span of the mice receiving treatment at younger age with mild phenotype. However, restoration of high levels of dystrophin expression failed to prevent disease progression to the mice receiving treatment when disease was already at advanced stage. The results could be critical for design of clinical trials with antisense therapy to DMD.
Once a dmd boy gets to a certain progression age it will be to late for exon skipping to stop the progression because the progression has already run it's coarse.It is insane to make them wait .I am guessing at age 15-17 would be the point of no return or maybe sooner?
Where is that trial at?..................................................................................I expect a FDA approval right after 24 weeks of new trial.