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Friedreich's Ataxia Center of Excellence Created with $3.25M Gift

NEW YORK (GenomeWeb News) – The Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence has been created with a $3.25 million gift from the Friedreich's Ataxia Research Alliance (FARA) in partnership with the Hamilton and Finneran families.

Friedreich's Ataxia (FA) is a progressive neurogenetic condition found in about one in every 50,000 people worldwide. No approved drugs currently exist for the condition, which is characterized by a progressive loss of coordinated movement and a loss of balance.

The goal of the new center is to work with pharmaceutical industry partners to develop drug candidates. Additionally, it will establish a biomarker development program; add cardiac expertise in FA research and clinical care; increase the capacity for and open more clinical trials; and create a dedicated drug discovery unit, the partners said.

The Friedreich's Ataxia Center of Excellence is co-directed by David Lynch, FA program director at Children's Hospital of Philadelphia, and Robert Wilson, a professor of pathology and laboratory medicine at the Perelman School of Medicine at the University of Pennsylvania.

The center continues a 16-year collaboration between Penn Medicine, CHOP, and FARA to improve FA patient care. In that time they have collaborated in research and clinical trials that have shed light on the metabolic dysfunction that underlies FA. Their work has also resulted in a database of well-document patients and more than 20 drug candidates. The FA clinical program at CHOP is the largest in the world, they said.

"Achieving a more holistic approach to both research and care is one main intent of our gift," FARA board member and donor Tom Hamilton said in a statement. "The Center will allow scientists and physicians to exchange insights regularly, not just as projects allow."

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