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Miracles on the Horizon!
crustpope:
First of all, thank you all so much for the prayers that were offered on Micah's behalf at Nationals. While I was unable to be there with you in person, it means so much to me to know that I am part of such a loving and caring community.
Recently, My wife and I have had some Amazing news that we would like to share with all of you
To help you understand what Muscular Dystrophy is, basically it is a degenerative muscle disease where all the muscles of your body (including your heart) slowly deteriorate due to the fact that an MD patient cannot produce (or does not produce enough) Dystrophin. Dystrophin is a protein that protects muscles from deteriorating. In an MD patient any muscle damage is basically muscle lost forever. MD patients usually lose the ability to walk on their own by age 12 and often die by the time they are 20. Usually cases are not caught until the child shows significant weakness around the ages of 5-8. My son's case was caught much early ( he is 1 and a half) because we were having him tested for something else and happened to stumble on this.
As far as Micah's news goes, short version is this: They are in the final stages of a treatment for Micah's version of Muscular Dystrophy. To explain it in detail gets pretty complicated but if you imagine Micah's X chromosome as a computer program, the part of his X chromosome that produces Dystrophin, a protein that protects muscles from deteriorating, is damaged. When the DNA is read, it gets to this point and crashes, producing no dystrophin. This lack of Dystrophin production is what causes his accelerated muscle deterioration.
In the successful treatment, they have inserted an engineered molecular plaster that helps the gene "skip" the damaged portion and still produce Dystrophin. This is called "Exon Skipping." In the first trials individuals had dystrophin production increase from 40 to 100%. while 40 doesnt sound like a lot, you only need about 36% to retain major motor function (read: be able to walk into your elderly years). The great news is that starting next month, the full scale human subject testing on Exon Skipping will begin at Cincinnati Childrens Hospital. While Micah is too young to be a candidate for the test, this test and its (hopefully) successful conclusion will be over long before He shows symptoms. Furthermore, I have it on good authority that given the fact that this is the FIRST treatment for MD, when it goes before the FDA for approval, it will be fast tracked and approved barring no major problems or complications are revealed in testing.
This is absolutely AMAZING News and has the MD community in a tizzy. Of course the medicine will be astronomically expensive but at least there will be a treatment option available instead of the long goodbye that many of those who suffer from MD currently have to look forward to.
I want to encourage all or you to continue praying for a successful testing and that we are all able to find ways of funding and paying for this treatment. I have enclosed some recent pictures of Micah and my family so you can have a face to pray for as you lift him up. Thank you all so much!
crustpope:
Pics in this post. Most are from Vacation this year at Holden Beach, NC
Looks like someone has a drool problem
Right now this is my all time favorite picture. Micah goes HIGH! YAY!
This is a great picture of all of us. Obviously not to hard to get him to smile!
Making him a Reds fan Early!
Prof Underwood:
Matt,
This is awesome news! I just checked out your link and am fascinated by the science behind all this, but most of all, as your friend I am just excited for the hope that this gives to you and your family!
God answers prayers :)
TheJaylor:
Wow, great news!
Jeremiah 29:11 "For I know the plans I have for you declares the Lord. Plans to prosper you and not to harm you, plans to give you a hope and a future."
This is the verse that ran across my mind when I read this and it make me think about how the Lord answers prayers!
crustpope:
Some pictures are up. @ Mark, the science behind this is brilliant. The "medicine" is delivered via a weekly IV drip and from what I understand, the molecular plasters cover up the damaged section of the DNA allowing the gene to be read with a blank spot instead of a damaged spot. This allowes the protein to be made, even if it is slighly less effective or produced at slightly less amounts.
Either way the end result is that MD patients show little to no muscle deterioration and should be able to walk into their advanced years.
Of course there is little stull understood about any possible long term consequenses so that still has to be considered, but from my prospective anything that allows my son to survive into his adult years is a step in the right direction!
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