Agada

FED recognized the need for robust clinical trials in mouse models of DMD. There had been a history of poorly carried out drug trials in rare disease mouse models, and FED wanted to turn this around, making DMD mouse research an example of rigor and good science. 10 years ago, FED convened the first international meeting of DMD mouse researchers in Washington DC to discuss the design and conduct of robust mdx mouse trials. FED and CDMRP then provided key funding to Dr. Kanneboyina Nagaraju to establish the first centralized mdx mouse clinical trial facility at Children’s National Medical Center. FED also provided key support to the TREAT-NMD group to work with Dr. Nagaraju to develop standard operating procedures, and an internet resource to ‘raise the bar’ on how robust mouse clinical trials should be done.

The Mouse Drug Testing Facility at Children’s National grew rapidly, carrying out over 50 drug trials in mdx mice for foundations, NIH, DoD CDMRP, universities, biotechs and pharma. The demand on the facility soon outstripped the capacity at Children’s National, and a new venture philanthropy spin-off was created in 2013 – AGADA BioSciences. FED continues to support drug trials in the mdx mouse at AGADA, with a focus on combination therapies of existing FDA-approved drugs in collaboration with DuchenneUK foundation.

To read publications of Dr. Nagaraju on the mdx mouse model, see here…….

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.