Genetic disorder center offers complete care

In an ongoing effort to combat neurofibromatosis, School of Medicine researchers have established the Washington University NF Center — a one-of-a-kind institute for collaborative and interdisciplinary research on these complex genetic disorders.

“Our goal is to develop effective therapies for individuals with NF,” said David H. Gutmann, M.D., Ph.D., center director and the Donald O. Schnuck Family Professor of Neurology. “To achieve this goal, we must understand the function of the NF genes in health and disease.”

The comprehensive center provides complete care for patients with NF, a complex genetic disorder that is more widespread than cystic fibrosis, Huntington’s disease, hereditary muscular dystrophy and Tay Sachs disease combined.

NF affects almost every organ system, causing a predisposition for tumors to grow on nerves in the brain and throughout the body.

The center’s mission is to galvanize and promote NF research, achieve significant breakthroughs in the diagnosis and treatment of nervous-system tumors, while establishing the University as an international beacon for NF research.

“Our team aims for a future in which early diagnosis will be followed by an accurate assessment of how an individual’s disease is likely to progress and the use of tailored treatments specifically targeted to that individual’s medical problems,” said Gutmann, also a professor of genetics and of pediatrics.

“These advances will not only benefit individuals affected with NF, but will also have significant impact on the treatment of children and adults with similar medical problems.”