Hey, when do u think there will be a cure? I just wanna hear ppls thoughts, honest, when do you think there will be a cure?

speculate.

Hey  --

I think there are lots of neat technologies out there that have good potential. I just wish the whole process moved along faster. I find myself more focused lately on finding something that stops progression vs. creating a reversible cure only because I'm content where I'm at even though my function is not that great.

But overall I think there will at least be a treatment in the next 10-15 years, hopefully sooner.

I've also heard estimates of around 10 years (5 to 10 years in my case), so slowing the progression of the disease and maintaining function is always a good idea of course.

Hey Guys

These are great questions. We are trying to have the best experts come to our annual conferences to answer these types of questions. It looks like this years BMD Conference will be in Los Angeles. So please try to make it. We all have questions like this and we had some very good discussions last year and even some human trial studies that were being done with BMD patients on treatments, rather than cures.

Kevin

I was poking around on Facebook the other day and came across the following article: http://quest.mda.org/news/mda-funds-utrophin-magnet-dmdbmd

Basically, it appears that this particular research institution is attempting to increase levels of utrophin where dystrophin is lacking as this may hold some benefit. Could be a useful treatment for all those with DMD/BMD disease.

On other fronts, I've heard a lot of encouraging stories. I am currently a veterinary student and have linked up with a muscle biologist in the animal science department whose main research is in muscle development and dystrophies. I heard from him a number of encouraging things:

1) Some mutations have been successfully treated in animal models using gene therapy, which utilizes adeno-associated viruses that are non-immune stimulating in dogs (a similar, non-reactive serotype is available for people) that can effectively "repair" the gene and produce more functional dystrophin.

2) Exon skipping drugs are currently undergoing clinical trials and have been shown to increase the amount of a truncated but still functional dystrophin protein in DMD patients, producing a syndrome more like BMD, which would be a huge leap for sufferers of that disease.

3) There are a lot of extremely bright people working on this disease. The individual I've been speaking with is the director of the Washington Center for Muscle Biology, a collaborative effort by up to 70 member labs from the University of Washington and Washington State University. This center helps streamline research by opening up all of the member labs and associated technologies for all members to use.

This summer I will potentially be participating in research funded by an MDA grant to develop protocols for evaluating exercise performance in wild-type and mdx mice (mice genetically engineered to have MD) so that scientists who have treatment protocols for MD have a robust model to test their treatments on before they are proposed for use in humans.

I will certainly keep you all updated as I hear anything new! Not only do I have BMD, but I also have access to world class research scientists who are doing just the research that we all need.

Andrew