Just saw their newest release today (July 30, 2009) showing BioMarin plans to start Phase I Clinical Trials for Utrophin first quarter, 2010. The release didn't say where they plan trials to take place.

Keep fingers, toes and everything crossed this one goes well !!!

cheryl

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Thanks for posting this Cheryl!

I noticed some AVI news also, they are moving to Seattle. http://www.avibio.com/pr/pr428.php
Thanks for this post. We all are hoping for this to happen!
You have just made my day, thank you for the post. I keep checking their site to see their progress & still stating pre-clinical so this is great news. Nadine :-)
Hi all,

I contacted BioMarin several times to follow up on when/where their trials would go. So far this proposed timeframe is all I have. But, I suspect if we ALL contacted them (squeaky wheel syndrome) they just might provide more information, so please e-mail or call them as soon as you can;
bpps@bmrn.com
and or
Rachael Gress, Medical Information
1-800-983-4587 medinfo@prosancorp.com

Please contact them to get more answers and let us know here what you came up with!

Thanks,
cheryl
I would like to see the news release too just so I know what I'm talking about when I call.
Okay I found it: http://www.medadnews.com/News/Index.cfm?articleid=642447 Go to the paragraph that reads: BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy:
BioMarin is completing formulation work and toxicology studies 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 replacement for the missing dystrophin protein
in DMD patients.
Hi Roxanne,

Did you try this email address:

bpps@bmrn.com - this one takes awhile to get a response or
for faster results try: medinfo@prosarcorp.com

Actually I called them this morning & spoke to someone who didn't know much but said she would find someone who could answer my questions, if there is an answer available to the public. They just now emailed me back stating no further information is ready at this time.

Perhaps we all should keep contacting them tho...something has to give sometime.

cheryl


Roxanne said:
I tried emailing them instead of calling b/c i'm currently @ work. my email is bouncing back, undiverable. I can't seem to locate an email address on their website as to where to address my questions.
Hi Roxanne-

Sounds like the same response I received. Standard verbage sticking within the thin margins of their "forward looking statements", which are geared for investors. When I got that same message (by the way was your message from a Dr Wells?) I e-mailed back saying I'm no investor, I don't wish financial harm on any company that might have a treatment or cure to save my child, and isn't there any other information he can offer.

I really don't expect to hear back. But even they should try to understand children with fatal genetic diseases should have priority for information! Hope I didn't piss them off.

Will you let us know if you hear anything else? I will.
cheryl


cheryl

Roxanne said:
rec'd a response to my inquiry. "BioMarin is completing formulation work and toxicology studies and expects to initiate a Phase I Trial by the first quarter of 2010" No additional information is available at this time. Lets hope they will have more info available as we go into the fall.
i think they are smart by not giving more information. one, they are protecting themselves in case something delays the start of a trial and, two, they aren't hurting families of Duchenne with the possibility of false hope. It saves them from getting all the emails/phone calls from families saying, "But you said you would start on this date...what the #$%@?"

Another thing to consider, after PTC wasted meaningful $'s in attorney fees to fight against a family who wanted the drug that was promised to their son, biotechs are probably going to be very cautious of the information they let out now.
Hi Christian,

Yea, I think they, as in the case of all pharma companies, are receiving legal advise on how much and what to say when. It was explained to me that they have interpreted a specific action or law (Paul can cite which one) in a certain way and therefore refrain from letting us know anything until after the fact. That specific law or action (not referring to the PTC case you mentioned) can be interpreted many ways. In other words, they can tell us more if they desire to do so and without fear of being sued. Their attorneys also know how this can be done, and how to go about it so as not to harm their company. They just don't bother.

Like you, I am all for all kinds of hope, even false hope can be beneficial in the short term. If BioMarin is planning a phase I early next year seems to me we are past the "false hope" section and on to the "organizing a clinical trial" part...unless they aren't organizing a trial... and their words are to be written off as "forward looking statements" (aka balderdash).

Parents of critically ill or dying children deserve a little more respect than that, I feel. Where are their ethics? If they could only tell us where they plan the trials to take place or some other minor bit of information, that would be better than keeping us in the dark until their attorneys allow them out to play.


cheryl



MicahsDaddy said:
i think they are smart by not giving more information. one, they are protecting themselves in case something delays the start of a trial and, two, they aren't hurting families of Duchenne with the possibility of false hope. It saves them from getting all the emails/phone calls from families saying, "But you said you would start on this date...what the #$%@?"

Another thing to consider, after PTC wasted meaningful $'s in attorney fees to fight against a family who wanted the drug that was promised to their son, biotechs are probably going to be very cautious of the information they let out now.
Ps:

I hope nobody out there is afraid of calling, e-mailing or contacting any pharma company. Like Christain said I doubt they ENJOY being pestered but they are for-profit companies. Businesses like to hear from their customers. If the phone isn't ringing - its a bad thing. It is the kind of "pestering" they wish for and the reason we see so many direct advertisements from pharma's, like Viagra commercials, on tv. Its why they have toll free numbers and receptionists. If a company can't handle a little pestering now and again I doubt they have the right constitution to conduct clinical trials.
cheryl cliff said:
Parents of critically ill or dying children deserve a little more respect than that, I feel. Where are their ethics? If they could only tell us where they plan the trials to take place or some other minor bit of information, that would be better than keeping us in the dark until their attorneys allow them out to play.

I couldn't agree more.

and...

cheryl cliff said:
Businesses like to hear from their customers. If the phone isn't ringing - its a bad thing. It is the kind of "pestering" they wish for and the reason we see so many direct advertisements from pharma's, like Viagra commercials, on tv. Its why they have toll free numbers and receptionists. If a company can't handle a little pestering now and again I doubt they have the right constitution to conduct clinical trials.

definitely...and to add on to that, they NEED to hear from us. If they hear from us, they know they are doing work for people who care and are in need of their work.

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