My BRAVE Story: We have two sons with Duchenne Muscular Dystrophy

We have two sons with Duchenne Muscular Dystrophy, James is 13, and Jacob, who has a dual diagnosis of DMD/autism is 9. James was recommended for a trial two years ago but learned that his specific mutation did not qualify. So we must continue to wait for the next exon skipping drug to begin trial stage and expect it to be many years before it will get approved by the FDA.

I fear the progression of this muscle wasting disease in our boys' bodies during the interim will reach the point of no return before they are allowed access to a treatment or better yet, a cure.

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