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.
He talked passionately about boys with muscular dystrophy that desperately need a new drug that those taking it believe works.Wish I had heard the whole interview.
Johnson railed about out of control FDA today on the Vicki Mckenna show on Milwaukee radio WISN. Johnson said the FDA is blatantly neglecting to act on the direction of congress. Ron wants people to have a "right to try"
google "mckenna right to try" and watch the videos.
The 4% threshhold prevented muscle damage altogether. Unfortunately the lower cutoff on the left side of the distribution was 3% (the median was 3.9%). It is hard to interpret a skewed distribution but it suggests that a continuum of protection exists below 3% where functional loss is slowed due to the protein production rate lagging the turnover rate.But this is old news published in 2013.
excerpt from Dumont, et al:
Dystrophin is expressed in differentiated myofibers, in which it is required for sarcolemmal integrity, and loss-of-function mutations in the gene that encodes it result in Duchenne muscular dystrophy (DMD), a disease characterized by progressive and severe skeletal muscle degeneration. Here we found that dystrophin is also highly expressed in activated muscle stem cells (also known as satellite cells), in which it associates with the serine-threonine kinase Mark2 (also known as Par1b), an important regulator of cell polarity. In the absence of dystrophin, expression of Mark2 protein is downregulated, resulting in the inability to localize the cell polarity regulator Pard3 to the opposite side of the cell. Consequently, the number of asymmetric divisions is strikingly reduced in dystrophin-deficient satellite cells, which also display a loss of polarity, abnormal division patterns (including centrosome amplification), impaired mitotic spindle orientation and prolonged cell divisions. Altogether, these intrinsic defects strongly reduce the generation of myogenic progenitors that are needed for proper muscle regeneration. Therefore, we conclude that dystrophin has an essential role in the regulation of satellite cell polarity and asymmetric division. Our findings indicate that muscle wasting in DMD not only is caused by myofiber fragility, but also is exacerbated by impaired regeneration owing to intrinsic satellite cell dysfunction.
I think there's also the possibility of contempt of congress, with the commissioner being arrested by the sergeant of arms and called before the house or senate.
Every medical institution operates under a set of policies and procedures that are managed by a governing body, eg an infection control committee. Every time a new treatment modality or equipment is brought in the policies and procedures must be created and updated, and staff needs to be trained. The production of blood products is a process involving many carefully controlled steps. Validation is the implementation of the new process that has been modified as a result of introducing intercept. An outcome standard is adopted and quality control processes are altered to assure the results are as expected. The reason is that any change in the process of producing blood products has the potential to introduce errors. How long this takes depends on the institution and the urgency. I would expect some q/a is called for in parallel to make sure the process works as expected before cutover.
The first thing is to execute; finish up the P3 and file NDA. There's also a cap rate requirement for nasdaq so the stock needs to get pumped up. A reverse split doesn't boost the cap rate by itself. Getting shorts to cover would go a long way. Another order from BARDA to make up for the portion given to chimerix would do the trick. It's a chicken and egg proposition gotta have good news to raise capital and gotta raise capital to remain intact. The wild card is Ron Perelman. It is entirely conceivable that he would loan money to pay the judgement.
capitulation. Why would the share price climb on huge volume with a negative adcom? The FDA is captured. Somebody knows this will get approved. It was the last chance to cover.
You are mistaking the outcome of a contentious law suit for management. How would you have managed things better, wise guy? You were here using a different alias with the same agenda back in the days of Cindy, Moskowchess, and rivalmoney. Why don't you grow up and accept the fact that you made a mistake (like the rest of us) and move on. Blaming somebody for your bad judgement is infantile.