Treat-NMD

The European Community has a long history of funding outstanding trans-EU network grants. Many of these have focused on rare disease researchers and the patient/family communities they serve. TREAT-NMD was initiated in 2007 through funding by one of the EC ‘calls’ for proposals to develop research and clinical infrastructure for rare disease. TREAT-NMD is coordinated by Newcastle University, and the outstanding neuromuscular research group housed there. TREAT-NMD has taken the lead internationally in developing standards of care (and translating these to dozens of languages), developing standard operating procedures for pre-clinical studies, coordination of patient registries internationally, and many other key roles.

EC grants are generally given for 5 years only, and the EC funding for TREAT-NMD ended in 2012. This created a vacuum of financial support for a very successful and critically important effort. FED and the CDMRP rose to the challenge of supporting TREAT-NMD in the time of transition to distributed funding sources, including providing key funding to the TACT group (TREAT-NMD Advisory Committee for Therapeutics).

FED continues to provide extensive support to the TREAT-NMD infrastructure.

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.