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Permalink Reply by David on January 25, 2015 at 1:54pm

If you can't read article it describes funding in the 100s of millions of dollars for 'personalized medicine' In the US. Seems to have bipartisan support (GASP!)
All the exon skipping would qualify I would think!

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Permalink Reply by Jason Darienzo on January 25, 2015 at 3:27pm

It would be great if some of the 'personalized medicine' dollars went to DMD.

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Permalink Reply by Maria Salamon on January 26, 2015 at 2:18pm

Much of the personalized medicine money does go to DMD however there is more than one type of DMD and there is no possibility for there to be a one-size-fits-all medicine, it is simply not in the capacity of modern medical science to do so at this point. It should have bipartisan support because no case of DMD should be treated the same as any other case of DMD.

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Permalink Reply by Andrew Kerr on January 27, 2015 at 11:14am

Isabella,

There are actually some treatments being developed that could help all kids with DMD, like myostatin inhibitors and utrophin up-regulators that should help all kids with DMD regardless the specific mutation they have.

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Permalink Reply by David on January 27, 2015 at 11:18am

When politicians announce something like this they often have the $$ but only short list of projects to fund. By the time it gets to congress, the more tangible projects the better. So, we need to act fast to convince congress that DMD treatments should be included, even though we already know not all DMD treatments would qualify.

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Permalink Reply by Maria Salamon on January 27, 2015 at 5:55pm

Oh, I understand, thanks for clarifying.

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