From: baw1064

To: dysferlin@yahoogroups.com

Sent: Thursday, July 21, 2005 11:49 PM

Subject: [dysferlin] Re: interesting article.

Thanks for posting the link, it is interesting, and it will be
exciting to see what the results of the clinical trial will be. 
Also someone pointed out to me that in a recent issue of Nature (I
belive it was July 14), there is an article about a study in which
MDX mice were given a chemical compound which plugs holes in the
cell membrane of cardiac muscle cells. 

What is interesting is that this compound does exactly what
dysferlin normally does in the body, and thus might be an even
better therapy for dysferlin deficiency than for dystrophin
deficiency (which is what the MDX mice have). Also, there doesn't
appear to be any reason (to me, anyway) why the effect should be
limited to cardiac muscle and not have the same effect in skeletal
muscle as well.

Of course, there's concerns about toxicity, side effects, dosage,
etc. so it's not ready for prime time yet.

A summary (which everyone can access) is at
http://www.nature.com/news/2005/050711/full/050711-16.html

The complete article requires a subscription, which I do have
through my work, and have read the article. The summary does give
most of the highlights.

Brad