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Sarepta Therapeutics, Inc. Message Board

  • hwsimpsonshero hwsimpsonshero Jun 18, 2013 10:17 AM Flag

    Follow-up letter to FDA now on Jett Foundation website

    Message posted by Christine McSherry. Pretty powerful stuff.

    Regardless of how you feel about the science or the quality of the study, the drug works. You'd be a fool to bet against the parents of those with DMD. They're getting AA for eteplirsen. (And they should.) Just a matter of when.

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    • Good stuff--AA is a done deal! Thanks, Hero.

      Sentiment: Strong Buy

    • Christine has used some very strong language in the meeting, things like "you are not the expert in DMD, we are". She definitely is a warrior mom and nothing can stop her. However, those lines will irk people and cause unnecessary friction and delay. This really stands out as I read her blog. I hope this is nothing but she definitely can take some pages from "How to persuade people".

    • I believe the activists know much more than they have disclosed at this time

      but separate investors making money with parent horror

      I believe investors are going to make a ton of money here, a ton

      but this has nothing to do with the HORROR of the entire process

      so lets assume FDA winks ok on the filing of the AA app very very very soon

      and lets assume you have a teenage boy with dmd

      and lets assume everything goes perfectly with manu ramp up and ultimate FDA approval of the AA app

      and then lets assume the drug is ready to be commercialized


      not a second can be wasted from here forward

      that is the reality

      its right on the edge for those teenagers

      the very parents who dragged this to the AA app filing finish line have teenage boys


    • It simply is true ,they really need to get this drug to all the kids asap.dragging feet for no reason is cruel ..

      any person with good sense and data can figure this out....

    • Segment 3 of 3 (This sums it up nicely.)

      You will see children being children in the next hour. But you must understand, this is not normal, not in Duchenne. Kids NEVER get better in Duchenne. Once they start on their decline, their is no stabilization, there are no outliers who can suddenly climb stairs, this does NOT happen. The drug is working, Jett needs it now and so do all those amenable.

      Sentiment: Strong Buy

    • Segment 2: of 3

      I have listened to the company presentations, and I know that you have the data set – and it would be easy for you to point to something and tell me that I don’t know what you have in that data, but I am here to tell you that you are not the experts on Duchenne. These parents and myself live with this disorder every single day and have been for years, for me – 12 years. Some of us have more than one child with Duchenne. WE are the experts on the progression and timeline of this disorder. Our Duchenne community, as you know, are full of very savvy parents. We know what Duchenne is suppose to look like – and we know when we are seeing the the curve begin to change. We are here today to tell you that you have everything you need to approve this drug, so all the others diagnosed with Duchenne, whose caregivers are feeling helpless right now, whose lives are at stake – have a fighting chance.

      While you are going to hear stories today about boys in the trial, those on eteplirsen, I am here to remind you that there are many who were not lucky enough to get into this trial…and that this trial, if granted accelerated approval, will allow industry to address others in the community. Those are the young men and women that I represent…those who have do not have a chance, those who will lose the ability to walk, feed themselves, and breathe on their own. They will succumb to an untimely death – most notably within the next 2 years. The same amount of time to produce the results you are about to see – this population can NOT wait another 2 years. If forced to go the more traditional route for approval, that will exceed 2 years. You have all the information you need, you have the support from the community and public you need.

      Sentiment: Strong Buy

    • Here it is, in two segments:

      What we said to the FDA, part 2

      June 18, 2013

      Why we are here…

      We are here to urge you to approve eteplirsen for those amenable to skipping exon 51, those with Duchenne. We are here as parents, but we also represent the Duchenne community – they have asked us to come here and they have supported our trip here. They want you to know that you have the community support behind you for this decision. We have also brought with us 180,000 electronic signatures. People from all over have signed this petition, urging you to approve eteplirsen. Please read the comments, many come from those who have lost someone to Duchenne, their stories are heartbreaking and deserving of your eyes.

      In August 2011, 12 boys were lucky enough to get into the eteplirsen trial and no one knew what to expect – but now we realize, their lives have been changed forever. Fast forward to June 2013 – 2 years later, and I want you to think about that timeline – 2 years later, we know that the drug is working. Every single day, these parents, are seeing little miracles, miracles that we would have never expected to see after more than 2 years of living with Duchenne. We know the natural history of Duchenne, we know it well; we have several published papers on the natural history – today I will be referring to the data published in the 24 month longitudinal data by Mazzone. This paper describes the decline, and the ability to predict a decline in 113 patients. The trial was designed specifically to help identify inclusion criteria for ambulant patients in order to minimize risk of patients who may lose ambulation within the time of the trial.

      Sentiment: Strong Buy

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