The three ERV posters are online in abstract form. Search by date, monday at 12:30. ERV was superior to LEV in infections resistant to LEV.
Treatment was effective, ie. non-inferior at end of treatment, but LEV outperformed ERV by the post-treatment period.
ERV needs to be dosed longer via IV before switching to oral.
biotech trading is intersectional. You need to know about trading AND biotech. I'm here to see if this price point represents a bargain. It is curious that the TCP problem is not universal. Saw this sort of thing once before (leukocytopenia) that was traced to one lab tech not flushing a central line before sampling. The circumstances suggest that there's something besides or in addition to the drug causing immune system activation. Also I think GSK is sensitive about rnai after their failure with drisapersen in DMD with prosensa. In that case there was marked induration at the injection site, rendering placebo arm unblinded and requiring dose adjustment to below the therapeutic window. "Once bit by snake always afraid of rope" . Larger P3 trials are very expensive. Critical question is whether the chemistry works without limiting side effects. It's an uphill battle if you have to co-administer steroids. It would raise the question of which agent is doing what.
I think the author deleted it because it was not producing the desired effect.
The 3% number comes form a study in knockout mice. The median level of dystrophin in the population was 4% and the minimum was 3%. It showed that in mice, greater than 4% rescued muscle. Where the levels were 3-4%, progressive deterioration was seen. The study did not say that levels below 3% are insufficient to prolong the age at which loss of ambulation and death occur. The study suggests that low levels, below 3% might prolong the loss of ambulation, but should not be expected to cure the condition - that would require 4% on average.
Margin requirements apply to customers of brokers. Others have a different set of rules. If you own the stock long you may short without any cash or margin reserves. However I've never had a brokerage account that allowed both. The capability is restricted for "retail" investors. Don't be mislead about shorts getting margin calls. Only the retail shorts have to sweat it out.
repackaging and relabeling. old lots will be combined to accomodate the doubled dosage. I suspect the additional doses are free, since they were sold on a per course basis.But if siga is doing the packaging, there is likely a contract change order. I recall contingent contract items for alterations due to shelf life and dosage.
I don't disagree that Farkas is dug in against FDASIA. There is a history behind the posture going back to cold remedies.
Reasonably likely standard dates back to quarantine policy. Its intent is to prevent arbitrary and capricious suspension of liberty.
It has too. Results published in Annals of Neurology:
Over a 3-year period, eteplirsen treatment markedly reduced loss of ambulation compared to matched historical controls. After 3 years, 2 of 12 (16.7%) eteplirsen-treated patients lost ambulation, compared with 6 of 13 (46.2%) historical control patients ≥7 years of age amenable to exon 51 skipping
The level of dystrophin necessary to preserve muscle in humans is unknown. Decades of research may be required to characterize the dose-response of dystrophin, independent of eteplirsen. In the meantime there is no question that some dystrophin is produced with eteplirsen, and that studies show slowing of deterioration with treatment. They have to reconcile the clinical observations ( clear benefit ) against the equivocal assays of dystrophin without knowing for sure how much is needed to make a difference at a biochemical level. I think this boils down to a question of basic science. The hypothesis is that if you can get the body to make dystrophin it will provide a clinical benefit. It is very clear to me that the the theory is correct. I think sometimes scientists get so wrapped up in the minutia that they overlook the obvious. Or to quote Bob Dylan "you don't need a weather man to know which way the wind blows".
read the paper. search "Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy" Published Jan 2016 in Annals of Neurology.
Not only did eteplirsen-treated patients experience a slower decline in 6MWT distance, fewer eteplirsen-treated patients lost ambulation than untreated, matched historical controls. During the 3-year treatment period, 2 of 12 eteplirsen-treated patients lost ambulation by year 1 and none thereafter (16.6%). In contrast, 6 of 13 (46%) historical control patients lost ambulation by 36 months.
Ataluren addresses 10% of DMD, in a disjoint segment not amenable to exon skipping. No overlap, thus no competition. Also its MOA is unknown. It does not provide "read through" according to follow on investigation.