HAROLD VARMUS, M.D.
DIRECTOR, NATIONAL INSTITUTES OF HEALTH
DEPARTMENT OF HEALTH AND HUMAN SERVICES
BEFORE THE
HOUSE COMMERCE SUBCOMMITTEE ON HEALTH AND ENVIRONMENT
MARCH 26, 1998

The story of Friedreich's ataxia illustrates how many areas of clinical and basic research can come together in unexpected ways. In this rare disorder, research involving neuroscience, genetics, clinical medicine, molecular biology, and even biology of yeast and bacteria are converging. The findings offer insights to basic biology and to many other disorders, and illustrate the importance of understanding the mechanism of disease in order to devise treatments.

Friedreich's ataxia now joins a growing list of degenerative disorders, such as Parkinson's disease, in which free radicals have been implicated. As with progress in many rare diseases, what we discover about cellular changes and therapeutic approaches in Friedreich's ataxia may lead us to important insights about more common disorders.