A $3.25 million gift from the Friedreich's Ataxia Research Alliance (FARA), in partnership with the Hamilton and Finneran families, established the new Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence.

Three longtime allies - Penn Medicine, The Children's Hospital of Philadelphia, and FARA, a nonprofit organization dedicated to curing Friedreich's ataxia (FA) - joined forces to create the center, which will work with pharmaceutical industry partners to develop drug candidates as well as biomarkers for FA.

FA is a rare, progressive neurogenetic condition characterized by progressive lack of coordinated movement and loss of balance. It also involves degeneration of heart muscle and nerve cells. Symptoms usually begin in childhood, and most patients are confined to a wheelchair by their mid-to-late 20s. Myocardial failure and/or arrhythmias are the most common cause of premature death.

"Integrating cardiac expertise into the care of patients is one major step forward this gift allows us to pursue," said Philip R. Johnson, MD, executive vice president and chief scientific officer at CHOP. "Rare diseases are often an area where philanthropy can make a difference, and the generosity of these donors will make a significant impact."

FARA, CHOP, and Penn Medicine have shared in research and clinical trials that have elucidated the metabolic dysfunction underlying FA. Their work has created a database of well-documented patients and a pipeline of more than 20 drug candidates ready to be mined for new therapies. Currently there are no approved drugs to treat FA.

The Friedreich's Ataxia Center of Excellence is co-directed by David Lynch, MD, PhD, FA program director at CHOP, and Robert B. Wilson, MD, PhD, professor of pathology and laboratory medicine at the Perelman School of Medicine. Drs. Lynch and Wilson both serve on FARA's scientific advisory board.

Additional information about the new Friedreich's Ataxia Center of Excellence is available here.