Personalized medicine for rare mutations READ THIS

Hello PPMD members and Pat,

Back in April, Pat mentioned in a post in the Duplications Group data base - that PPMD was pursuing projects with Prosensa and another one would focus on "personalized medicine specifically focused on rare mutations."   Pat, you also state that PPMD needs help financially with this.

I have asked several times in emails to Pat about details back in April, May and June - about what the personalized medicine with rare mutations was.  I have never gotten an answer.  Does anyone have any details on this?  Pat, can you share any details of this alliance that you speak of?  Since the myostatin clinical trial was put into a holding pattern as well as exon skipping is only for exon 50 or 51, I am very much without alot of hope.  As parents with a son with DMD, hope helps us through the valleys.  I would appreciate any information that parents or Pat would share.  


Thank you,

Char Burke

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This Site is the only one I could find with some information that was informative, but it was dated in 2009.


Having two little grandsons with duplications, I would also like to know the details about the "personalized medicine with rare mutations" that Pat is referring to.  We all need hope to cling to, especially those of us who have sons or grandsons with rare mutations.  Perhaps during or after the conferences, more light will be shed on this. 

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