So Dystrophin replacement or production of'' is the primary fuction of repairing or correcting.
June 9, 2015 10:32 AM EDT ... Roth Capital analyst Debjit Chattopadhyay
reiterated a Buy rating and $45 price target on Sarepta Therapeutic (NASDAQ:
What was fixed was all the public pressures that drove the fda crazy and now all is good... The rest is ok to put out there ''substance news for Sarepta is required by sec.
These boys were chosen because they were expected to have a decine per natural history and age releated.drisa was quite the other way ,much younger kids that would not show a decline based on age and natural history facts. fyi
CAMBRIDGE, MA--(Marketwire - Oct 3, 2012) - Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its lead exon-skipping compound, eteplirsen, met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT) over the placebo/delayed treatment cohort in a Phase IIb extension trial in Duchenne muscular dystrophy (DMD) patients.
Eteplirsen administered once weekly at either 30 mg/kg or 50 mg/kg for 48 weeks (n=8) resulted in a statistically significant increase (p≤0.001) in dystrophin-positive fibers to 47.0% of normal. The placebo/delayed treatment cohort, which had received 24 weeks of eteplirsen at either 30 mg/kg or 50 mg/kg following 24 weeks of placebo (n=4), also showed a statistically significant increase in dystrophin-positive fibers to 38.3% of normal (p≤0.009).
"These data represent a significant milestone and a defining moment of progress and hope for patients with DMD and their families, as well as for those of us in the scientific community who have been pursuing potential treatments for this devastating and deadly disease for decades," said Jerry Mendell, M.D., Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb study. Dr. Mendell added, "By addressing the underlying cause of DMD, eteplirsen has demonstrated unparalleled effects on enabling dystrophin production and slowing the progression of the disease as measured by the 6-minute walk test, with no treatment associated adverse events. While eteplirsen is targeted to DMD patients with a specific genetic mutation, I think the implications for all DMD patients with related genetic mutations are clearly evident."
Eteplirsen administered once weekly at 50 mg/kg over 48 weeks resulted in an 89.4 meter benefit compared to patients who received p
Fda deal, stop the parents from public outcries and stop chris and maybe we can move forward professionally. Notice all the parents stopped..you cannot make the fda look bad or you will pay. Sarepta on a great coarse now ...fyi... Look for super fast approval from here.
Show me how drisa is producing any positive drug effects? If you care so much why havent you done good research? You will put kids on drisa for 5 years and they decline and never get that health back again.! Drisa is dosed in low doses because it is toxic and that is why it fails, if they could dose higher it may work but they can't.etep works because doses are higher and it is non-toxic.. '' 2 different types of drugs''
1. Giving a drug that gives zero benefits will cause dmd boys to lose something they may never get back''quality of living''
2. Why would a drug company offer false hopes to kids that may never get the chance to walk or play longer when they know the drug they have is worthless..''?'' it really shows the true value of that comapny!
A total of 186 boys were randomised to this double-blind, placebo-controlled study (DMD114044) and received drisapersen at a dose of 6mg/kg/week (N=125) or placebo (N=61) via subcutaneous injection over 48 weeks. The difference in 6MWD (mean (CI) 10.33m (-14.65, 35.31), p=0.415) between drisapersen and placebo groups did not reach statistical significance. There was no treatment difference in key secondary assessments of motor function: 10-meter walk/run test, 4-stair climb and North Star Ambulatory Assessment. The most commonly reported adverse events included injection site reactions (78% for drisapersen vs 16% for placebo) and renal adverse events (including subclinical proteinuria; 46% for drisapersen vs 25% for placebo). No patients had thrombocytopenia.
Full evaluation of the benefit-to-risk profile of drisapersen treatment across all studies is anticipated to be completed by year end. This may include analyses of pooled results from various drisapersen studies.
"We appreciate that these results will be disappointing for boys with DMD and their families. We would like to sincerely thank all those who participated in the study for their commitment," commented Carlo Russo, Senior Vice President, Head of GSK Rare Diseases Research & Development. "We are committed to evaluating the outcome of this study in the context of the overall development programme with experts in the field, and we expect such evaluation to help inform our next steps for drisapersen. It is our hope that progress will be made in an effort to help boys with DMD."XXXX----=======
XXX... Duchenne muscular dystrophy patients treated with GlaxoSmithKline's (GSK_) experimental drug drisapersen have been hospitalized due to kidney toxicity and low platelet counts, according to a Glaxo scientist who spoke at a research meeting in Rome last Sunday. Glaxo has disclosed some safety data from early-stage drisapersen clinical trials previously, mainly related to moderate cases of proteinuria (excess protein in the urine) and decreased thrombocytes, which play a role in blood clotting. However, the significance of the adverse events attributed to drisapersen, also known as GSK-2402968, escalated with Sunday's disclosure of hospitalized patients. Dr. Rohit Batta, global medical leader in Glaxo's neuromuscular rare disease unit, said four Duchenne muscular dystrophy (DMD) patients treated with drisapersen required hospitalization due to thrombocytopenia and that "several" patients with "severe proteinuria" also required hospitalization. Batta's remarks were made during a presentation on Sunday, Feb. 24 at The XI International Conference on Duchenne Muscular Dystrophy. The conference was held in Rome and organized by Duchenne Parent Project Onlus, an Italian DMD advocacy group. Batta provided no other information about the hospitalized drisapersen patients during his presentation. A Glaxo spokesperson contacted Wednesday confirmed Batta's remarks and added the DMD patients hospitalized were all enrolled in the ongoing phase II and phase III clinical trials of drisapersen. The hospitalizations had occurred "over the last two years" and had been discussed at previous medical meetings.
Drisa was in a trial that had all boys that were so young ''kids that would not have a decline''misleading the fda''during the last phase of the trial those boys got a little older and decline showed up and proved too be a very unworthy drug.facts''on top of it all drisa would be a nightmare for long term use based on toxic outcomes.
you will see down the road what i mean......do your DD ..