Hope

When parents are told that their child has a disease that is as awful as Duchenne Muscular Dystrophy, there are plenty of reasons to be despondent. After all, this is a disease that is “incurable,” and largely known as “untreatable.” It has only been through relatively recent advances in palliative care and a growing body of knowledge about substances such as steroids that kids are growing into their twenties with the disease. No major drug company has ever made an investment in Duchenne Muscular Dystrophy, because despite being the world’s top genetic lethal childhood disease, it is still classified as rare – one out of 3,500 boys. Biotechs are just now beginning to “kick the tires” out of interest in a potential profit in Duchenne Muscular Dystrophy treatment. The overwhelming majority of funds raised in the name of benevolence for Muscular Dystrophy go toward care, not toward research. And the governmental commitment to Duchenne Muscular Dystrophy, while improving, is woefully inadequate for the challenge.

Why, then, are we filled with so much hope? The reason is that Duchenne Muscular Dystrophy is a disease with a simple biology. Flaws on one gene one protein. We’ve had our gene identified for 17 years. Thanks to the work of Dr. Hoffman and many others throughout the world, we know a great deal about exactly how – and why – this disease progresses. You can’t say that about heart disease, cancer, diabetes, or any of the other diseases that receive billions of dollars in research funding. And the simple reality is that several teams of researchers have already identified ways to stop this disease in its tracks in the laboratory.

What separates us from the marketplace? Only two things: time, and money. They are everything.

Leadership

Board of Directors

Joel Wood, President | Scott Sinder, Secretary | James Wood, Treasurer | Dan Carson, Director | Dave Heil, Director | Brad Todd, Director

Advisors

Dr. Eric Hoffman Dana Wood Monica Heil Tina Carson

Staff

Michelle Payne, Executive Director

Financial & Tax Information

FED strives to be transparent in our work. Here are links to our most recent IRS form 990 and audited financial statements. If you have questions, please do not hesitate to contact us.

What is Duchenne Muscular Dystrophy?

The words “incurable” and “untreatable” have been closely associated with Duchenne. It is an X-chromosome disease that afflicts boys, and it will never be eliminated through genetic screening because the dystrophin gene is the largest gene in body – times ten – and thus much more prone to have flaws that are not inherited. The trajectory of this disorder is insidious. Duchenne boys typically are not diagnosed until they are four years old or older. They tend to get better in the so-called “honeymoon” stage, which usually lasts until the early teenage years. But their muscles are deteriorating and cannot regenerate. Dystrophin is part of the chemical glue that holds muscle cell walls together, and those walls eventually collapse and are filled with collagen that causes fibrosis. First the legs give out, slowly, then, the arms and upper body. The disease moves from skeletal muscle to smooth muscle tissue. Lungs and hearts deteriorate. Corticosteroid treatment forestalls the muscle deterioration, but steroids have terrible side effects such as making bones brittle. Unlike many single-gene-defect disorders that only strike at certain ethnicities or demographics, Duchenne is a disease that does not discriminate – it is the world’s leading lethal inherited disease, afflicting one in 3,500 boys. The incidence rate among all races is identical. As the world becomes more conscious of issues of racial disparity, it is clear that standards of care are resulting in disparate quality of life and certainly life expectancy among sufferers of different races. You get the picture. Duchenne Muscular Dystrophy is excruciating, ruthless, sadistic, and painful. More than anything, it is cruel. The FED’s mission is to identify, leverage, and bring to market treatments that will benefit ALL sufferers of this disease.