Hot off the press

This is a brand new article, independent research, not related to BioMarin.

Dr Wiclox update

It is on my side bar, but just in case you missed it... Read this very interesting article research article about finding another therapeutic agent in addition to CNP (which Biomarin is testing).

For the nerds, here is the detailed published study by Dr Wilcox (of Cedars Sinai) and collaborators. It is not easy reading, but I can guarantee you will learn a lot.

Dr Wilcox on Biomarin drug for hypochondroplasia

Growing Stronger organised an LP doc talk a while back and the following question was asked of Dr Wilcox, who is one of the scientists behind the BioMarin drug. I am sure I have shared this link before, but to get a full picture of how involved he exactly is, I suggest you read  the April 5, 2012 blog post on this blog, sorry the direct link to the post doesn't work.
"What are your thoughts about the potential of treatment of hypochondroplasia with BioMarin's drug in trial?"

You can listen to his answer here at 7:02

I tried to transcribe as well...

"Yes the CNP should work fine on that. Biomarin isn't studying it first as there is less of them and they would need a less growth promotion, so they would probably need a lesser dose. I don't think they will be doing a trial for the drug for HCH in the near future. It may not be until the drug is approved. I wouldn't hold your breath for the next 3 years. It would take that long until it's available for hypochondroplasia.

BioMarin phase 1 update

OK, here we go:

3 things strike me (other than the general shock of how little testing pharmaceuticals need to do for an orphan drug before they move into phase 2... , but I go through every time I look into this)
1. No other adverse reactions shared other than the jump in the blood pressure just like in the animal trial.
2. Then the  "We are presently evaluating options for further development in various forms of achondroplasia." I presume this probably means hypochondroplasia and thanatophoric dwarfism.
3. The reference to the "non-growth endpoints" - that is an interesting consideration I haven't thought of before, lucky I am not in the business of developing drugs...

ACH growth study

All right, here is the official link so the growth study which will precede the Biomarin BMN-111 phase 2 clinical trial has started recruiting. I suppose everyone has speculated that other skeletal dysplasias which are caused because of changes of FGFR3 behavior might benefit from the treatment as well... (look at the patent description here), it is very obvious that these will not be included in the initial trials, as HCH is listed as an exclusion criteria.
Not a reason not to follow the future developments though...

More presentations

Here is the link where all Growing Stronger presentations can be easily found. They are all well worth watching.

Update on the BioMarin clinical trial

Without any comment, worth reading: http://www.growingstronger.org/drs-wilcox-and-krejci-blog.html

Bloomberg article re BioMarin trial

I love the perspective of this article:

http://www.bloomberg.com/news/2012-01-23/biomarin-s-dwarfism-medicine-marks-growth-of-400-000-rare-disease-drugs.html

The clinical trial has really started!!!!!

Phase 1 of the clinical trial has begun.

http://phx.corporate-ir.net/phoenix.zhtml?c=106657&p=irol-newsArticle&ID=1662147&highlight=

I also asked a question of the geneticist who is involved with the trial if they think the drug will be used for HCH as well as ACH and as I expected, the answer is yes... YAY!!!

The clinical trial is starting

I am extremely excited about this

http://growingstronger.org/wp/category/blog

I know it could be 5-10-15 years, but at least they are working on developing a drug to help bone growth...

Here are 2 really cool videos, the first one is an overview of ACH, the second one is about the drug tested:

http://www.youtube.com/watch?v=3ZVGnbGpPmU&feature=player_embedded

http://www.youtube.com/watch?v=5EeKi1xYXas&feature=related