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left2rightdoor 671 posts  |  Last Activity: Jul 30, 2015 7:18 AM Member since: Jul 16, 2012
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  • left2rightdoor by left2rightdoor Jul 30, 2015 7:18 AM Flag

    More traffic accidents among teenagers than the world has ever seen before. I was just in a car climbing a hill behind a fiat going 45 uphill on the freeway and when I went past looked over and a kid had his phone at the 12 position on his steering wheel and was trying to steer while texting

  • Trying to look at Panama Mexico penalty, and all I get from Yahoo is audio of an ad I can't even find on
    the page to shut off, and it turns out to be John Kasich running for President. Another guy I can forget
    based on Yahoo political advertising slowing down the internet.

  • Kaye talked about publication of natural history data for the DMD boys, to come before a fall
    advisory committee meeting. The publication of AVI Marburg Phase I data is merely a lagniappe.
    The main information package is yet to come.

  • Because YHOO - has bad (terrible) search suggestions; too many promoted results making it difficult to get to real search results; and slow scripts loading causing browser to stop or abort.

  • Reply to


    by pebble16x Jul 17, 2015 4:59 PM
    left2rightdoor left2rightdoor Jul 17, 2015 5:15 PM Flag

    But what is the Ex-AABACO date. Nov 30? Dec 5? Nov 15? When will they give the date to hold for the
    spinout. Someone should ask MM at the conf call on Qtrly earnings. Everything else pales in relation to the spinout ex-date.

  • left2rightdoor left2rightdoor Jul 17, 2015 3:01 PM Flag

    Change 2 letters with eteplirsen approval
    Going from SR to IC, ie ICPT like market cap.

  • In May and June, CDC guidance on H5N1 said that stockpiled antivirals including peramivir could be used in human cases of H5N1 infections (probably in view of multi- state widespread poultry outbreaks) and in the guidance for clinicians updated as of June 11 include peramivir as among the recommended antivirals.

  • left2rightdoor by left2rightdoor Jul 7, 2015 12:16 AM Flag

    FDA has particular guidance for how to label drugs under the accelerated approval pathway. It should be
    clear that approvals are conditional based on continuing confirmatory trials, and that there is uncertainty. However, for many DMD patients and families, they will already know all about "reasonably likely" benefits of Eteplirsen

    Posted 24 March 2014
    By Alexander Gaffney, RAC
    A new guidance document published by the US Food and Drug Administration (FDA) is intended to clarify how drugs approved through the accelerated approval pathway should be labeled.
    FDA's accelerated approval pathway is one of several tools used to expedite the approval of medicines intended to treat serious or unmet needs.
    Under Section 506(c) of the Federal Food, Drug and Cosmetic Act (FD&C Act), accelerated approval is reserved for products intended to treat or cure a "serious or life-threatening condition" based on surrogate endpoints that are "reasonably likely to predict clinical benefit."
    For example, if a drug is intended to treat a deadly type of cancer and early data indicate that it has slowed the progression of the disease, it could be eligible for accelerated approval.
    In addition to the severity of the disease, FDA is also supposed to take into account its rarity as well as the availability (or lack thereof) of alternative treatments. If ample FDA-approved treatments are already available, a new drug is unlikely to obtain accelerated approval unless it demonstrates a marked improvement in safety or efficacy relative to the existing treatments-a "meaningful advantage over available therapy."
    It's also important to note that drugs approved through the accelerated approval pathway are given somewhat tentative approvals. "Approval under this section will be subject to the requirement that the applicant study the drug further, to verify and describe its clinical benefit, where there is uncertainty as to the relation of the surrogate endpoint to clinical benefit,

  • Someone is posting falsehood that no Eteplirsen treatment in Europe. the exon 53 skip is ongoing in Europe.
    posting materially false information knowingly creates legal liability

    4053-101: PHASE I/II STUDY

    Official Title: A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

    Purpose: This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.

    Enrollment Status: This study is recruiting participants at clinical sites in Europe.

    More information: Identifier: NCT02310906

  • Reply to

    Comparing NDA's

    by speedbll Jun 26, 2015 1:26 PM
    left2rightdoor left2rightdoor Jun 26, 2015 2:33 PM Flag

    That's what caused FDA skepticism, delayed DMD drugs for 2-3 years. Only now if FDA internal opinion recovering to believe in dystrophin. Time for a make-up call on Eteplirsen by the umpires who blew the first 5 innings. Has already started with rolling NDA, may accelerate in next month.

  • So someone is still interested in this clinical trial as of April 21, 2015. Maybe a surprise airburst one of these days.

  • left2rightdoor by left2rightdoor Jun 22, 2015 11:39 PM Flag

    Genocea 52% red in viral shedding. vical 19%
    Genocea 48% reduction in genital lesions. vical 51%
    Not clear why lesion reduction comparable or better, shedding less.
    worth looking at some more data.

  • left2rightdoor left2rightdoor Jun 22, 2015 9:39 PM Flag

    The PTC/BMRN had a table by an academic, not a rep of either co, showing how many exons were in clinical trials, Sarepta had 7, 2 more than anybody else. Sarepta, as Ed Kaye said at PPMD Connect Conference, is committed to getting therapy to as many treatable DMD boys as it can. SEVEN exons is more important than a lot of handwaving about 6MWT

  • because the regulatory path is too long. Go get em Ed.
    PPMO versaility and specificity (Limited off target effects !!!!!!)
    Using patient reported outcome measures
    Regulatory approval pathways Accelerated Approval and Fast Track

  • left2rightdoor by left2rightdoor Jun 19, 2015 2:17 PM Flag

    If it worked for 12 patients, and it is shown safe for additional 20-30 patients, and. clear evidence shows that a drug produces a biochemical response that mitigates the serious disease then the benefit risk balance is positive.

  • Ed Kaye starts w/Sarepta at 2:40, then PTC, Biomarin

  • left2rightdoor left2rightdoor Jun 17, 2015 9:30 AM Flag

    Extends cash runway Last presentation Jun said $111 cash, expenses 65-80/yr, funds to mid 2016. Upfront $33 covers them to end 2016, NDA for HAE. Stockpile would be on top of that. BCX 4430, new targets BCXxxxx coming.

  • Reply to

    PPMD conference this week

    by simp08801 Jun 15, 2015 8:08 AM
    left2rightdoor left2rightdoor Jun 15, 2015 11:38 AM Flag

    National End Duchenne Rally on Capitol Hill Steps, followed by meetings with legislators. 9-12:30 June 19
    see PPMD Connect Conf Agenda

  • Reply to

    PPMD conference this week

    by simp08801 Jun 15, 2015 8:08 AM
    left2rightdoor left2rightdoor Jun 15, 2015 11:12 AM Flag

    Interesting session could. be FDA Farkas on Jun 19 1:45 - can't say much specific but reemphasize FDA commitment to getting therapies to DMD community as soon as reasonably likely to show benefit , this after DMD demonstration on capitol steps in morning

  • left2rightdoor left2rightdoor Jun 14, 2015 12:48 AM Flag

    See PPMD's comments about Biomarin getting rolling NDA filed on April 27
    Suggests drug approval by 1Q 2016. A credible pathway, although always uncertain, is -mid year NDA complete, 3Q Advisory Committee, FDA approval within 6 months after AdComm.

    ParentProjectMD's insight:
    The Duchenne community should celebrate. Today, Biomarin submitted the rolling NDA on drisapersen. This means we now have a second NDA submitted for Duchenne. Santhera is likely to follow soon. By first quarter 2016, we may see 3 approved drugs with more to follow. This is exactly what we have been working for, hoping for.

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