BioMarin announced the start of their Phase I clinical trial, which has already begun, in healthy volunteers for utrophin upregulation. They expect results 3rd Qtr 2010. Not sure where this trial is taking place tho...

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Good news that they were able to respect the timeline!


http://www.marketwatch.com/story/biomarin-initiates-phase-1-clinica...
Great news. 2010 will be our year.Let us hope and pray for the BEST.
Keep in mind that there are 3 companies looking at Utrophin Upregulation. Prosensa will be next, but they are still 12-18 mths away from a phase 1. This is AWESOME news!!!! I am so excited and with a 9 mth turn around on phase 1, should make 2011 a GREAT year!
Hi Lori,
Which is the other company for utrophin upregulation ( apart from Bio Marin and Prosensa)?
Prosensa is NOT working on utrophin upregulation, PTC does as part of project catalyst (utrophin upreg and myostatin inhibitors are 2 possible drugs they would like to bring to trials).
That is wonderful news Cheryl .... Deb and I were talking about the SMT C1100 trials (for anyone that was not aware BMN 195 was developed by Summit, and renamed by Biomarin) yesterday as their timeframe for preclinical had blown out so badly. (Summit were predicting phase 1 to start mid 2009, but have now updated their website following the Biomarin press release) Here's hoping they are ultra efficient at getting the results out, so phase II can begin ASAP.

Parents the world over will sleep a lot better tonight knowing we are taking steps to help ALL boys. Thankyou for sharing this milestone with us all :)
I don't recall right now....I just remember there being 3 at conference...maybe it is PTC, prosensa and Biomarin. Ofelia is right below, PTC is working on one and THAT is the one that PAT was talking about being 12-15 mths away. Prosensa is exon skipping....but aren't they doing a utrophin too? I certainly can be wrong on that!

RAKTIM SINGH said:
Hi Lori,
Which is the other company for utrophin upregulation ( apart from Bio Marin and Prosensa)?
I think the third utropin upreg would be Justin Fallon's stuff (Biglycan). We have not heard about that in some time. I'm not sure how "close" he is to clinical trials though...

Lori Ware said:
I don't recall right now....I just remember there being 3 at conference...maybe it is PTC, prosensa and Biomarin. Ofelia is right below, PTC is working on one and THAT is the one that PAT was talking about being 12-15 mths away. Prosensa is exon skipping....but aren't they doing a utrophin too? I certainly can be wrong on that!

RAKTIM SINGH said:
Hi Lori,
Which is the other company for utrophin upregulation ( apart from Bio Marin and Prosensa)?
That sounds righ Ofelia....thanks!

Ofelia Marin said:
I think the third utropin upreg would be Justin Fallon's stuff (Biglycan). We have not heard about that in some time. I'm not sure how "close" he is to clinical trials though...

Lori Ware said:
I don't recall right now....I just remember there being 3 at conference...maybe it is PTC, prosensa and Biomarin. Ofelia is right below, PTC is working on one and THAT is the one that PAT was talking about being 12-15 mths away. Prosensa is exon skipping....but aren't they doing a utrophin too? I certainly can be wrong on that!

RAKTIM SINGH said:
Hi Lori,
Which is the other company for utrophin upregulation ( apart from Bio Marin and Prosensa)?
Thanks for posting this. It has made my day. I have been waiting for this news.
You are more than welcome Amrit!! It made my day too :) I was so overjoyed I thanked the nice people at BioMarin and wished them godspeed.

Amrit said:
Thanks for posting this. It has made my day. I have been waiting for this news.
Great news! Thanks for posting.

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