I remember when Chris G. said it was ways around that ruling and now i know how.I also know now it is 3-ways around Biomarin's bad drug for dmd.
anyone with any good thinking skills can see this plainly...
A real trial is when you put boys on the drug that you are expecting a decline''sarepta'' not like biomarin/prosensa/gsk did ''real young boys you don't expect a decline from...misleading trial.
On top of that cake these kids were older by far than any of prosensa kids...
After 48 weeks of treatment, eteplirsen administered at either 30 mg/kg or 50 mg/kg for 48 weeks (n=8) resulted in a statistically significant increase (p d 0.001) in dystrophin-positive fibers to 47.3 percent 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 37.7 percent of normal (p d 0.008).
Results from the study suggested that at least 12 weeks of treatment was needed to observe increases in dystrophin production in muscle biopsies. In addition, there was no meaningful difference in dystrophin production between the 30 mg/kg and 50 mg/kg dose arms at 48 weeks.
Walking Ability: A key secondary endpoint and the study's principal clinical outcome measure was the 6-minute walk test, a standard and well-accepted measure of walking ability and clinical function in DMD.
After 48 weeks, patients in the 30 mg/kg and 50 mg/kg dose cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) showed a statistically significant treatment benefit of 67.3 meters (p d 0.001) when compared to the placebo/delayed-treatment cohort (n=4). This difference exceeds the 28 to 44 meter treatment effect reported in clinical studies that have served as a basis for the FDA approval of treatments for other neuromuscular disorders.
The children that is on it will lose valuable health that they will never get back and that is a horrible waiste.this is a real fact and i hope the fda sees this.
please show me a link where it shows production of dystrophin that is of benefit
The only prosensa/biomarin patent allowed was exon 44 and 51''17 percent of dmd boys but
you can skip other exons close to the 51 and 44 and will do as good with dosing adjustments.Also it is under appeal .'' My professional opinion is that the patent should be based on mechanism of action of the exon / how can you patent a gene or a exon? Sarepta and Biomarin has two different drugs that would be different mechanisms of actions?
dystrophin production''drispersen has no information on producing any dystrophin that i can find anywhere?The dosing is so low due to toxic problems you cannot get what you need //hiding a fact like this is shameful based on hurting children's health and lost health .
The drug, called drisapersen, was unable to improve walking distances compared to placebo in the study, which involved 186 boys with a mutation in the dystrophin gene thought to be amenable to treatment with the antisense oligonucleotide.
After 48 weeks, boys who received drisapersen could walk approximately 10m further than those on placebo, but the difference was not statistically significant.
Earlier results from a phase IIb trial revealed a significant, 35-metre improvement on the 6MWT for drisapersen compared to placebo after 28 weeks' treatment and in June it was awarded breakthrough status by the US FDA.
Drisapersen is designed to induce 'skipping' of exon 51 when the dystrophin sequence on DNA is written to RNA, with the aim of producing a truncated but semi-functional form of the dystrophin protein that is abnormal in DMD, restoring some muscle function.
GSK and Prosensa's drug not only failed to improve scores on the six minute walking test (6MWT), the phase III study's primary endpoint, but was also unable to confer a benefit of secondary measurements of motor function, including the 10-meter walk/run test and four-stair climb.
"We appreciate that these results will be disappointing for boys with DMD and their families," said Carlo Russo, head of GSK's rare disease
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.
somewhere.................................or just some good research teams doing common sense dd