Anybody have new updates on any research? Seems like the forum has been pretty quiet lately.

Hi,, there are only two trials right now that may help PPL with BMD... One trial is now opened in Canada.  Here's the website.

http://www.acceleronpharma.com/content/products/ace-031/duchenne.jsp

the other is biomarins Uptrophin.. that's currently opened in the EU.. They are currently conducting a trial for safety.. we should hear something more towards the 3Q of this year.  

Think that's about it..  Most research is to help those affected by duchenne MD,, trying to turn duchenne into becker..

Roxanne.

Here's a good summary of all the research presented at the latest PPMD conference in Denver. It seems like there are some promising new approaches that are nearing human trials.

Attachments
 Abstracts.pdf [1092 KB] ::

http://www.acceleronpharma.com/content/products/ace-031.jsp

how close are we to an effective treatment? ... :(

Hi,, after the conference this weekend you may have a clearer picture as to where we are with future treatments.  I personally feel we are still years away.. I had high hopes for Biomarin's Utrophin but it turns out this drug is not effective..90% of drugs fail during phase I & II so Biomarin's recent news confirms these numbers.   I'm only 3 1/2 years in & already fit up with the highs & lows of BMD research

unfortunately could not attend the conference...

I will post videos from the conference, later this week. There were three promising avenues of research pitched at the BMD meeting. One is in human safety trials now, one is recruiting for human trials, and one is probably a year away from human trials. Overall, some good news and some action. We will see how they all shake out.

Kevin

thanks Kevin.. Would Santhera be one of those trials you are speaking about.. they are currently in phase III for Duchenne. It may also offer some benefit to ppl with BMD.

SOrry forgot to add the link.. here it is..

http://www.santhera.com/index.php?mid=111&vid=&lang=en

Hey Roxanne

None of the new compounds presented was Santhera's Idebenone. Idebenone is essentially a super duper free-radical vaccuuming compound that is already available today. I've heard of some cases where it was helpful, but they seem scattered and mostly in other forms of non-DMD and non-BMD M.D. . I'm glad it's being looked at formally in a study setting to see what it really can do (or not do).

The three presentations at BMD2 were three new pathways and drugs that target whole new approaches to the BMD/DMD problem. I think they represent a diversity of methods and approaches which is a good sign. Overall they scientists and researchers were extremely energized by the conference, so I hope we'll have even more presentations and pitches next year.

Kevin