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OK - I'm confused...if the entire population of DMD patients is considered, isn't that an argument against orphan designation?
I have always understood that the primary reason for giving DMD orphan status is that it's actually an infinite number of possible deletions and mutations, each manifesting it self in uniquely nuanced way.
What am I not getting here?
OK - I'm confused...if the entire population of DMD patients is considered, isn't that an argument against orphan designation?
I have always understood that the primary reason for giving DMD orphan status is that it's actually an infinite number of possible deletions and mutations, each manifesting it self in uniquely nuanced way.
What am I not getting here?
It seems that the development of any drug for DMD will take at least 10 years,so I am afraid that the DMD boys who may benefit from any treatment (if any) ARE NOT BORN YET!!!!!!!!!!!!!!!!!!!!
Idebenone is still in a clinical trial!notice that Idebenone is just an antioxidant and it is available as supplement,so immagine how long it is gonna take a drug as ACE 031 or Utrophin (I will not say gene transfer because it is gonna take 200 years!!!!!!
I am very disappointed!!!!
I work for a medical device/pharma company. We have devices that are drug coated that have over 70 different combination of sizes. All 70 didn't have to go through the clinical trial process. What happens is companies have to prove equivalancy accross the matrix. So they take say 3 sizes/combinations and show that they work. Then all 70 combinations are approved. I don't have the lingo to develop a protocol for exon skipping in such a manner, but there must be someone out there who can?? Imagine a trial that will have 3 exon skipping patches that say are aimed at exon 10,30 and 50? And then get approval for a multiple of patches? I know what people are going to reply.... well each patch is in effect a different medicine..etc etc... but....the Reg bodies have to change....or ...lets just let generation after generation after generation after generation suffer and die!!
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