Biomarins Utrophin Upregulation phase 1 trial slated for first quarter of 2010

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-- BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy:
BioMarin
is in the final stages of preparing the regulatory filing and expects
to
initiate a Phase I trial by the first quarter of 2010. BMN-195 is an
orally available small molecule which may upregulate utrophin, a
potential substitution for the missing dystrophin protein in DMD
patients.
http://www.reuters.com/article/pressRelease/idUS228413+28-Oct-2009+...

[Oct 29, 2009 1:17:24 PM]

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I'll be there too...I don't know how many times I have wished this upon myself and not on him =), I can totally handle being a guinea pig if it helps these boys...
My understanding is that the exon skipping drugs and PTC 124 had to be used on DMD boys first because they were expected to cause Duchenne Muscular Dystrophy if administered to healthy individuals, who would then start producing out of frame dystrophin proteins if exon 51 were deleted, or if their final, and correct, stop codon were read through and the dystrophin protein would continue to be constructed until by chance a randomly created pseudo stop codon would be triggered somewhere after exon 79. I think there is no reason to believe that high levels of utrophin in an otherwise healthy adult might be harmful.
That does make sense....

Paul Cliff said:
My understanding is that the exon skipping drugs and PTC 124 had to be used on DMD boys first because they were expected to cause Duchenne Muscular Dystrophy if administered to healthy individuals, who would then start producing out of frame dystrophin proteins if exon 51 were deleted, or if their final, and correct, stop codon were read through and the dystrophin protein would continue to be constructed until by chance a randomly created pseudo stop codon would be triggered somewhere after exon 79. I think there is no reason to believe that high levels of utrophin in an otherwise healthy adult might be harmful.
Just saw this Cheryl: Will call them tonight when I get home from work.

cheryl cliff said:
You can reach BioMarin at:

415-506-6700
105 Digital Drive
Notato, Ca 94949

If they sigh you up Lori & Donna let me know :) I'll be there too!!
Ok Donna. I also called them and had to leave a message. I've contacted them before and it does take a little time for them to respond. Probably they will try to call me while I am away this week. I'll let you know and please share what ever information you happen across :)

Jacobs Mommom said:
Just saw this Cheryl: Will call them tonight when I get home from work.

cheryl cliff said:
You can reach BioMarin at:

415-506-6700
105 Digital Drive
Notato, Ca 94949

If they sigh you up Lori & Donna let me know :) I'll be there too!!

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