Duchenne Muscular Dystrophy – Severe Muscle Degeneration Disease

Integrated Oncology Network CEO and cofounder Jeffrey Goffman serves as the board chair of CureDuchenne, a nonprofit organization with headquarters in Newport Beach, California. Jeffrey Goffman and his fellow board members help lead the organization in offering support to individuals with Duchenne muscular dystrophy (DMD) and spearheading research efforts for a cure. A devastating muscle disease, DMD affects nearly 20,000 young boys across the United States and over 300,000 worldwide.

DMD is the most common and severe genetic disorder within the muscular dystrophy family. Defects in the genes responsible for producing a vital muscle protein cause the disease, and affected children usually receive the diagnosis before age 5. By the age of 12, patients lose enough leg muscle to become wheelchair bound, and most do not live past their mid-20s. Although it primarily affects boys, a small number of girls may also receive the diagnosis.

Defective genes prevent the body from forming the dystrophin protein, which leads to the progressive degeneration of muscles in nearly every part of the body and eventually compromises heart and lung health. Additional health complications include brittle bones, predisposal to pneumonia and heart failure, and the possible decline of cognitive functions. Patients can also develop fibrosis in the connective tissue between shortened muscle fibers.

CureDuchenne’s research strategies focus on providing support for new treatments to reduce DMD’s severity, drug repositioning, and protein replacement therapies. Since its establishment, the organization has advanced seven projects into human clinical trials and leveraged over $100 million from pharmaceutical companies and government agencies to fund cure research.

For additional information about DMD and the CureDuchenne organization, visit http://www.cureduchenne.org.

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