Why Duchenne Muscular Dystrophy is OUR Disease

Recently, the DMD community has had reason to hope. Sarepta, the manufacturer of eteplirsen; a promising drug to treat Duchenne, was encouraged by the FDA to submit a New Drug Application(NDA.) On November 12, the FDA abruptly changed their guidance stating an NDA filing for eteplirsen would be premature.  Sarepta has met every benchmark since the trials began.The results have been remarkable, not only showing improvement, but in some cases a reversal of symptoms. Duchenne Muscular Dystrophy is fatal. Period.

Why does this battle belong to a mother whose child has Hypermelanosis of Ito? In the heart of every parent of a child with DMD is hope. It is the same hope that is in my heart for HI. It is the same hope that is in your heart for your loved one, no matter the disease.

The rare disease community not only has to deal with the struggles surrounding a disease most doctors don’t understand, but we fight against government, corporate, medical and social indifference. That struggle is OUR war.

We are all busy fighting for our loved ones. We are all searching for treatments and cures. We are all watching children lose their lives. Why should we take the time to help the DMD community when our own children are running out of time? Because a victory for DMD is a victory for the entire rare disease community. Every battle that is won brings us closer to the end of the war. We are all allies. When given an opportunity to help another rare disease group, we must help. Please click on the following link to see how you can help the DMD community; http://jettfoundation.org/blog/how-you-can-help-give-them-more-time/.

 

I did not write this because I care for someone who has a child with DMD, though I do. I did not write this because my heart is broken for these families, though it is. I would do the same for any rare disease. I write to you because, despite living with the uncertainty all rare diseases bring, I know beyond any doubt we all need each other.