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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Another FANTASTIC bit of DMD news for us in America. My understanding, such as it is, is this trial is to be held on healthy volunteers. Phase II, I think will be for DMD boys.
Keeping fingers crossed on this one since it may help all boys-my son included, Jacob too Donna!!

:)
Cheryl, if that is right...phase one only on heathly volunteers (and I remember that being said from Conference 2 years ago), then they also said that that would make the phase 1 trial go faster. Which, of course, is GREAT! I will sign up if they need me too! I mean, I have PLENTY of fat that can be converted to muscle after all!!!! I will be more than happy to donate on behalf of our boys!!! Don't mean to make light...this is FABULOUS news...

cheryl cliff said:
Another FANTASTIC bit of DMD news for us in America. My understanding, such as it is, is this trial is to be held on healthy volunteers. Phase II, I think will be for DMD boys.
Keeping fingers crossed on this one since it may help all boys-my son included, Jacob too Donna!!

:)
I hope they consider opening the trails for DMD boys as well. I am not sure why this extra step.
I don't think this is an extra step. THis is the phase 1 for safety. It will be quicker and easier to try this on healthy individuals vs. DMD boys. If they prove safety, they can go to the phase 2 and at that time include our boys.

Bains said:
I hope they consider opening the trails for DMD boys as well. I am not sure why this extra step.
Hi Bains,

My understanding, again such as it is, is that doing clinical trial phase I's on healthy individuals is normal protocol. What is unusual is starting a clinical trial (phase I) using actual patients...especially children, as we have seen with Exon Skipping trials held in the UK. This phase I by BioMarin won't be considered an extra step, it is the first step toward approval for Utrophin upregulation with DMD boys.

Pray to god everyday this stuff works!!

Bains said:
I hope they consider opening the trails for DMD boys as well. I am not sure why this extra step.
Thanks Lori,Cheryl. I was not aware of that. I hope and pray that this stuff works.
i know that phase 1 for PTC124 was on DMD boys....

Bains said:
Thanks Lori,Cheryl. I was not aware of that. I hope and pray that this stuff works.
God I hope this works. Wyatt can only benefit from this, exon skipping wouldn't help because of his extra large deletion 18-41.
Interesting for those with splice site mutations as well.
Oh yea, PTC did do phase I on DMD boys. Keep fingers, toes, eyes, everything crossed that this stuff works Lori!!

Lori Ware said:
i know that phase 1 for PTC124 was on DMD boys....

Bains said:
Thanks Lori,Cheryl. I was not aware of that. I hope and pray that this stuff works.
I'm with your Lori! Where do we sign up!

Lori Ware said:
Cheryl, if that is right...phase one only on heathly volunteers (and I remember that being said from Conference 2 years ago), then they also said that that would make the phase 1 trial go faster. Which, of course, is GREAT! I will sign up if they need me too! I mean, I have PLENTY of fat that can be converted to muscle after all!!!! I will be more than happy to donate on behalf of our boys!!! Don't mean to make light...this is FABULOUS news...

cheryl cliff said:
Another FANTASTIC bit of DMD news for us in America. My understanding, such as it is, is this trial is to be held on healthy volunteers. Phase II, I think will be for DMD boys.
Keeping fingers crossed on this one since it may help all boys-my son included, Jacob too Donna!!

:)
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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