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Scientific Advisory Board

The Scientific Advisory Board serves as advisors on FARA’s scientific research priorities, goals and programs as well as medical issues that arise from FA and treatment implementation.

Key Responsibilities:

  • A commitment to FARA's mission; advancing treatments & improving clinical outcomes for individuals with FA.
  • A commitment to helping FARA grow and nurture the FA research community.
  • To promote collaboration in the FA research community.
  • Reviewing grants and making funding recommendations
  • Assessing gaps in research portfolio and making recommendations for new opportunites/research directions


Dr. Bronya Keats
Emeritus Professor of Genetics, LSU Health Sciences Center
FARA, Chief Research Officer

Dr. Giovanni Manfredi
Professor of Neurology and Neuroscience
Weill Cornell Medical College


Dr. Sanjay Bidichandani
Section Head of Genetics, University of Oklahoma College of Medicine
Department of Pediatrics
FARA, Board of Directors

Dr. Joy Cavagnaro
Access BIO

Dr. Joel Gottesfeld
Professor Emeritus
Department of Molecular Medicine
Scripps Research Institute

Dr. Sidney Hecht
Director, Biodesign Center for Bioenergetics
Arizona State University

Dr. Arnulf Koeppen
Professor of Neurology and Pathology
Albany Medical College

Dr. David Lynch
Professor of Neurology
Children's Hospital of Philadelphia / University of Pennsylvania

Jane Larkindale, D.Phil.
Executive Director
Duchenne Regulatory Sciences Consortium at
Critical Path Institute (C-Path)

Dr. James McArthur
Venture Partner
RA Capital Management, LP
FARA, Board of Directors

Dr. Marek Napierala
Department of Biochemistry and Molecular Genetics
University of Alabama

Massimo Pandolfo, MD, FAAN, FEAN
Professor (Clinical)
Department of Neurology and Neurosurgery
McGill University

Dr. Mark Payne
Professor of Pediatrics (Cardiology)
Indiana University School of Medicine

Dr. Helene Puccio
Department of Neurobiology and Genetics
Institute of Genetics and Molecular and Cellular Biology

Dr. Bernard Ravina
Chief Medical OfficerPraxis Medicines

Dr. James Rusche
Independent Consultant
Pharma and Biotech Product Development
FARA, Board of Directors

Dr. Kevin Whittlesey
Vice President, Translational Medicine
4D Molecular Therapeutics

Dr. Robert Wilson
Associate Professor of Pathology and Laboratory Medicine
University of Pennsylvania

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