The Foundation to Eradicate Duchenne (FED) raises money to support the world’s only clinical trials network for Duchenne Muscular Dystrophy (DMD). The clinical trials network known as CINRG works with scientists all over the world on therapies for DMD – a fatal disease which scientists now say is tantalizingly close to being cured.
For now, this clinical trials network prolongs the trajectory of the disease until a cure can be found. Funding this clinical trials network and the scientists with whom it works means that every dollar is critical in this race against time. The FED puts an overwhelming majority of the money it raises directly into research, which means that each dollar raised brings the boys suffering from DMD closer to the cure.
In pursuit of the cure, FED has raised millions of dollars for the best DMD scientists and doctors around the world. Their efforts have launched clinical trials with drugs such as prednisone, deflazacort and pentoxifillin – drugs upon which Duchenne boys now rely to slow the progression of the disease. These trials have been made possible by a number of small fundraisers and the opportunities for Congressional intervention that they have fostered. Consequently, federal appropriations and private fundraising by the FED have produced nearly $50 million in funding for DMD research.
This funding has gone to scientists across the country and throughout the world in order to advance DMD research through development of non-hormonal corticosteroids, gene therapy solutions and many more including “exon skipping”. This technique would allow the flawed gene to “read through” and create dystrophin where previously it could not. If successful, it could produce a genetic process that could change DMD into a much milder, and thus less fatal, form of muscular dystrophy. This approach is another that could be available in mere years, rather than decades.
Managing all of these efforts from his lab at Children’s National Medical Center in Washington, DC is Dr. Eric Hoffman. As a member of the team 17 years ago that discovered the gene that causes DMD, Dr. Hoffman well understands the stakes involved in this deadly disease. Presently, his work involves identifying the pathways of DMD that could create multiple new targets for medical intervention that could dramatically slow the progression of this disease. In addition, he serves as the lead scientific advisor to these fundraising efforts in coordinating these and many other scientists throughout the world. FED is making all of this happen as a result of personal contributions and fundraising events. With FED’s efforts, over $10 million in private donations has been raised to date. Scientific advancement is progressing as rapidly as the FED can fund it with new breakthroughs being discovered every day. Every dollar counts, as each goes to expanding the foundation’s efforts, adding new clinical trials and basic research, and bringing thousands of boys closer to the cure.
Despite all the successes and major scientific breakthroughs, time is of the essence, and this research can only continue to progress if there are funds to support them. While the best scientists in the world agree that a cure for this disease is inevitable, the only unknown is whether it will arrive in time for today’s sufferers of DMD.