I have to say...
I am impressed with the quick response, although it should be quickly addressed, so many times you sit and wait for a response.
Here we go...
Debbie
Dr. Miller here- I am the CEO of Edison. Thank you for your email below.
We have a call with the Seattle Children's hospital today to hopefully
finalize getting started.
We are 100% supportive of having EPI-743 available at Seattle Children's.
You can be certain of this. You need not in any way convince Edison or
myself of urgency or petition on behalf of your son or others. We were
founded by physicians and parents of children with mito disease with one
goal clinically meaningful treatment. While I will never second guess
what it is like to have a child with mito disease, I can tell you we are
heart felt in our mission.
Emails like this and others focus our attention even more and that is a
good thing. I will email you back after our call today and set
expectations on both the going forward process and time lines.
Thank you so much for taking the time to get in touch with us.
Best regards/g
Guy Miller, MD, PhD
Chairman, CEO
Edison Pharmaceuticals, Inc
5 comments:
Debbie,
I just emailed Dr. Miller as well. We will likely have to start seeing Dr. S in Seattle with Garrett in the next years. I am SICK (no pun intended) of seeing our kiddos fade while there IS a drug that is helping greatly with so many!!!!! I will let you know what type of response I get as well. I also asked that he let me know when he thinks use of the drug will expand past the "90 days to end of life" window the FDA has it stuck in as we have an adult friend slipping quickly who has been turned away for not being "sick enough yet." But that is government, not Edison. And don't EVEN get me started on my government bandwagon....not pretty!!!!!
Hugs to beautiful Hudson!! And I have more bracelets if you want some!!
Great big hug,
Clara-Leigh
I've heard through the grapevine that Edison will be expanding the use of this drug in the very near future. Right now they are running something called a "treatment protocol". This is for the kids that either based on their specific mito diagnosis or their current symptoms (this is where the 90 days til end of life comes in...which is also a very subjective term), they feel these kids need the most urgent treatment and therefore every kid gets the drug. What they are going to be switching to is a standard double blind study format (something they HAVE to do in order to eventually get this drug approved by the FDA)...this is good in that it will open the door and many more children will have the opportunity to get treatment...what is bad (especially if it's your child) is that some kids will not get the EPI-743, they will instead get placebo! It has to be done and it's a necessary evil, but when your kid has mito, you don't really care and you just want them to get the real medication!!! All of this being said...as of now this is not some miracle drug that will cure children of having mito. But, for some, it does appear to lessen symptoms and potentially (something we all hope for) extend life expectancy. It's not a cure all...but it's all we've got!!
I emailed him too. And now I am crying with this response. What a man with character. So hoping the drug gets there quickly.
Thanks for your info Tara, I appreciate any and all insight as I get bits and pieces.
I get this is not a cure by any means. I also know that nothing has worked to stop Hudson's seizures, and I have been told it can improve brain function, which for Hudson, he suffers so much in that area.
Since anti epileptic drugs don't do squat, I'd really like to see if this drug could potentially change that for the better for Hudson.
When nothing works....you grasp onto something that you hear is working for some, maybe it will work for my kid? I know it may not.
I also know a child who was enrolled at Stanford and passed away while being on the study....SO.....it is clearly not the answer, but there is some hope in it, and that is what we have to hang on to for now! Getting it into Seattle is the first step, and that is what I am trying to push for....
YAY! As they say, "The squeaky wheel gets the grease." Great job Momma! :) Hugs!
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