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FARAFARA Cure FA

Center of Excellence in FA

FARA-sponsored Research Initiatives
The Children’s Hospital of Philadelphia Center of Excellence in Friedreich's ataxia


FARA and The Cure FA Foundation have partnered together to launch the Children’s Hospital of Philadelphia (CHOP) Center of Excellence (CoE) in FA. The CoE in FA is a translational research and clinical care center dedicated to developing treatments and a cure for FA. It is comprised of a synergistic team of four core facilities, each focused on bench-to-bedside research efforts that together provide a holistic approach to realizing a cure for FA.

The CoE in FA has expanded to integrate in the FA space researchers and clinicians with like interests. This includes Dr. Shana McCormack, an endocrinologist at CHOP who is interested in understanding how mitochondrial dysfunction in FA and other mitochondrial disorders influences overall bodily functions, such as food and energy metabolism, as well as growth and development.



Clinical Research Core
(PI: Dave Lynch, MD, PhD)

The CoE in FA is a world leader in informing and executing clinical trials through interrogation of natural history data, developing and validating clinical endpoints, and patient reported outcomes.


Biomarker Discovery Core
(PI: Ian Blair, PhD)


The Biomarker Discovery Core works to identify and develop of blood biomarkers of FA disease progression, cardiac risk, and therapeutic gain with treatment intervention in FA patients.






Drug Discovery Core
(PI: Rob Wilson, PhD)

The drug discovery core of the CoE in FA works to identify novel drug targets through high-throughput screening. The core also tests and validates these targets in models of disease, with a focus on quick translation to human studies. Lastly, the drug discovery core works to improve industry-academic partnerships to enable rapid and efficient promising drug candidates.


Cardiac Clinical and Research Core
(PI: Kim Lin, MD)


The CoE in FA is a multidisciplinary care facility working to improve outcomes for individuals with FA. The CoE has a focus on cardiac clinical care and research.







 
 
 




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