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InterMune, AŞ Message Board

musherga 45 posts  |  Last Activity: 14 hours ago Member since: Apr 19, 2007
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  • Reply to

    Statistical Question

    by hlmaz2015 15 hours ago
    musherga musherga 14 hours ago Flag

    read the paper. search "Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy" Published Jan 2016 in Annals of Neurology.

    excerpt:
    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.

  • Reply to

    Is There Room For Two

    by roger1260 15 hours ago
    musherga musherga 15 hours ago Flag

    Been discussed. It addresses a different kind of mutation.

  • Reply to

    motley article on ptct

    by rxkell May 19, 2016 4:57 PM
    musherga musherga May 19, 2016 9:35 PM Flag

    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.

  • Reply to

    Senator Ron Johnson

    by musherga May 13, 2016 8:43 PM
    musherga musherga May 15, 2016 2:19 PM Flag

    thursday 5/12

  • Reply to

    Senator Ron Johnson

    by musherga May 13, 2016 8:43 PM
    musherga musherga May 14, 2016 8:01 AM Flag

    Thanks. This deserves its own topic.

  • Reply to

    "We don't understand how this drugs works"

    by duwanee111 May 14, 2016 4:31 AM
    musherga musherga May 14, 2016 7:28 AM Flag

    Sounds like you've been reading about statins.

  • Reply to

    Senator Ron Johnson

    by musherga May 13, 2016 8:43 PM
    musherga musherga May 13, 2016 8:59 PM Flag

    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.

  • musherga by musherga May 13, 2016 8:43 PM Flag

    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.

  • Reply to

    From Joining jack

    by thigrlsrk May 12, 2016 7:26 PM
    musherga musherga May 13, 2016 8:18 PM Flag

    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.

  • Reply to

    Drop Test

    by fgtins Apr 23, 2016 5:30 PM
    musherga musherga May 13, 2016 7:37 PM Flag

    dead cat

  • musherga by musherga May 4, 2016 9:53 PM Flag

    ultimately approved. prolongs life by a few months.

  • Reply to

    Lookout Woodcock, Dunn and Co!

    by michaelwebbseattle May 4, 2016 5:10 PM
    musherga musherga May 4, 2016 9:24 PM Flag

    Jurney vs MacKracken. An assistant secretary of commerce was sentenced to 10 days in jail.

  • Reply to

    Lookout Woodcock, Dunn and Co!

    by michaelwebbseattle May 4, 2016 5:10 PM
    musherga musherga May 4, 2016 9:17 PM Flag

    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.

  • Reply to

    validation

    by musherga May 4, 2016 9:05 PM
    musherga musherga May 4, 2016 9:11 PM Flag

    So for example how can one be assured that the blood was treated before uv exposure and not after?

  • musherga by musherga May 4, 2016 9:05 PM Flag

    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.

  • Reply to

    uplisting

    by delmarloop Apr 18, 2016 2:28 PM
    musherga musherga May 3, 2016 12:04 PM Flag

    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.

  • Reply to

    Short attacked.

    by jamesn13 May 3, 2016 9:59 AM
    musherga musherga May 3, 2016 10:51 AM Flag

    I think mm took out their stops. Shares must be getting hard to borrow.

  • Reply to

    Volume

    by greyzone513 May 2, 2016 12:21 PM
    musherga musherga May 3, 2016 10:09 AM Flag

    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.

  • Reply to

    LOL! 6200 shares

    by drod4ball Apr 27, 2016 5:12 PM
    musherga musherga Apr 30, 2016 8:50 AM Flag

    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.

  • Reply to

    CNBC last night

    by heavyphil Apr 30, 2016 7:02 AM
    musherga musherga Apr 30, 2016 8:36 AM Flag

    I'd wait for a double bottom.

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