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
Keep in mind that any good analyst,investor can see that any way you chop it up Sarepta has the drug of choice long term.It matters to me for one reason '' putting kids of Biomarin's drug for dmd will waist good years of health from those boys''
Genes contain codes, or recipes, for proteins, which are important biological components in all forms of life. In 1987, the protein associated with this gene was identified and named dystrophin.
DMD occurs because the mutated gene fails to produce virtually any functional dystrophin. (Individuals with Becker MD genetic mutations make dystrophin that is partially functional, which protects their muscles from degenerating as badly or as quickly as in DMD.)
''My long term research brings me to believe that Sarepta's science behind these drugs for dmd will be the start of correcting these mutations.''If you invent sometime you start somewhere and improve as you learn more.This is the start of a great drug platform that will avail in the future.
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: