Understanding Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy pic
Duchenne Muscular Dystrophy
Image: WebMD.com

A philanthropist and CEO in the healthcare management sector, Jeffrey Goffman serves as chairman of the Board of Directors with CureDuchenne. In his leadership role, Jeffrey Goffman helps the nonprofit organization in its efforts to find life-saving treatments for Duchenne muscular dystrophy.

Muscular dystrophy represents not a single disease, rather it functions as a category encompassing several genetic conditions with progressive symptoms related to muscle atrophy. Some muscular dystrophies begin in adulthood, while others begin in childhood. Duchenne muscular dystrophy is a childhood disease that almost always affects boys.

Symptoms of Duchenne muscular dystrophy can start appearing in infancy and often get worse with time. Muscle weakness, a hallmark of the disease, usually begins in the lower extremities. Eventually, children with the condition lose their ability to walk and may develop breathing as well as cardiovascular problems by early adulthood. Moreover, Duchenne muscular dystrophy may cause intellectual disabilities.

Though there is no cure for the disease, researchers are working on gene therapies, some of which are in early human clinical trials.

Advertisements

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.