Duchenne and the Mission of CureDuchenne

After receiving his BS in accounting from the University of Hartford’s Barney School of Business, Jeffrey Goffman earned his certified public accountant license and spent 14 years in public accounting. Currently the chairman and CEO of Integrated Oncology Network, LLC, an oncology management services and operator venture he started in 2008, he serves on the Board of Affiliated PET Systems, LLC, a firm he co-founded in 2001. Jeffrey Goffman generously supports several charities, including CEOs Against Cancer, Wings for Crossover, and CureDuchenne.

The most common and severe form of muscular dystrophy, Duchenne typically occurs in boys, with initial symptoms appearing around age 2 or 3. The muscle weakness that characterizes the disease is caused by a loss of dystrophin, a component of muscle tissue that gives muscles their stability. As boys with Duchenne grow older, they grow weaker, and even the simplest tasks become increasingly more difficult. They are usually confined to a wheelchair by age 12 and rarely live past their mid-20s, because by then the heart and muscles that regulate breathing become too weak to function.

There are currently about 300,000 boys with Duchenne worldwide, about 20,000 of whom live in the United States. First identified in 1986, the disorder’s mechanics have been extensively investigated. Gene replacement therapies, RNA-based technology, and pharmacological interventions all show promise as therapeutic approaches. Despite this, there’s still no cure for the disease.

CureDuchenne was founded in 2003 by the parents of a boy who was diagnosed with Duchenne. When he was diagnosed, they learned that because so few boys have the disorder—about one in 3,500—it doesn’t command a great deal of attention within the medical and pharmacological research industries. They established CureDuchenne, whose mission is its name, to raise public awareness of this devastating disease.