PPMD has recently posted PDFs from the conference presentations recently in San Diego. Link is at the bottom of this message.

In the exon-skipping presentation, on page 23, there was a statment about the recent AVI trial results that surprised me. The context is some possible reasons why after 6 months of treatment there was little or no clear evidence of clinical benefit.

"Study includes older patients, so it may be harder to get dystrophin expression with less muscle left"

This indicates that exon-skipping may not work for DMD sufferers who have reached a certain level of muscle loss. That's the first time I've heard of such a concern about exon-skipping.

  • Does anybody have any other information that discusses this matter? I do not find anything in mouse or dog studies which suggested the more advanced cases benefitted less.
  • Would this same concern apply to other treatments too, or is this something specific to exon-skipping?

I realize there can be no definitive statements about such efficacy, but it is incredibly important to my family to know if the science for one treatment predicts a drop in efficacy for older patients, while another treatment may not.

http://www.parentprojectmd.org/site/PageServer?pagename=Connect_con...

 

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Exon skipping is only one part of a cure. We will need a combination of drugs.

  • To slow and/or stop deterioration rate use Exon Skipping and Utrophin up-regulation
  • Use anti-inflammatory and anti-fibrosis to support muscle regeneration
  • Stem cells and Myostatin inhibitors to increases muscle mass

 

 

Richard,

I can't thank you enough for your succint explanation of the science here. It was precisely what I did not understand.

-David

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