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Team
Clinical Investigator Team
Jose E. Abdenur, MDChief, Division of Metabolic DisordersChildren’s Hospital of Orange County Piero Barboni, MDFounderStudio Oculistico d'Azeglio Enrico Bertini, MDHead, Unit of Muscular and Neurodegenerative DiseasesBambino Gesù Children's Hospital, Rome, Italy Francis G. Blankenberg, MDAssociate Professor of Radiology & PediatricsLucile Packard Children’s Hospital, Stanford University Medical Center Valerio Carelli, MD, PhDAssociate ProfessorUniversity of Bologna School of Medicine Carlos Filipe Chicani, MD, PhDVisiting Assistant Professor of OphthalmologyDoheny Eye Institute, University of Southern California School of Medicine; Federal University of Sao Paulo, Brazil Bruce H. Cohen, MD, FAANDirector of Pediatric Neurology and Professor of PediatricsAkron Children’s Hospital Salvatore DiMauro, MDLucy G. Moses Professor of NeurologyColumbia University College of Physicians Carlo Dionisi Vici, MD, PhDHead, Division of Metabolic DiseasesBambino Gesù Children's Hospital, Rome, Italy Gregory M. Enns, MB, ChB1Associate Professor of Pediatrics; Director, Biochemical Genetics ProgramLucile Packard Children’s Hospital, Stanford University Medical Center Laurel M. Fisher, PhDAssociate Director, Clinical Studies DepartmentHouse Ear Clinic, Inc. Stephen P. Hersh, MD, DLFAPAProject DirectorThe Marriott Mitochondrial Disorders Clinical Research Fund Michio Hirano, MDProfessor of NeurologyColumbia University Medical Center Elwood W. Hopkins, MDCaptain, US NavyUS Naval Hospital, Bremerton Stephen L. Kinsman, MDAssociate Professor of Neurosciences, Division of Pediatric NeurologyMedical University of South Carolina Kenneth C. Kubis, MDCaptain, US NavyUS Naval Hospital, San Diego Diego Martinelli, MDResearch Collaborator, Division of Metabolic DiseasesBambino Gesù Children's Hospital, Rome, Italy Susan L. Perlman, MDClinical Professor of Neurology; Director, Ataxia Center and HD Center of ExcellenceUniversity of California, Los Angeles Alfredo A. Sadun, MD, PhD1Flora Thornton Chair of Vision Research,
ProfessorDoheny Eye Institute,
University of Southern California School of Medicine Russell P. Saneto, DO, PhDAssociate Professor Neurology and PediatricsSeattle Children's Hospital, University of Washington William H. Slattery III, MDDirector, Clinical Studies DepartmentHouse Ear Clinic, Inc.

Corporate Team and Advisors
Guy Miller, MD, PhDChairman, CEO
Adjunct Clinical InstructorEdison Pharmaceuticals
Stanford University School of Medicine Martin J. Thoolen, PhDChief Drug Development OfficerEdison Pharmaceuticals Keith TsukimuraSenior Director & Controller, FinanceEdison Pharmaceuticals Lorraine GilmoreDirector, Office of the CEOEdison Pharmaceuticals Carl BergBoard of DirectorsEdison Pharmaceuticals Forrest SawyerBoard of DirectorsEdison Pharmaceuticals Taro InabaBoard of DirectorsMitsui & Co. Venture Partners III, LLC Pat N. Confalone, PhDVice President, Global R&DDupont Peter P. Giannousis, PhDSenior ConsultantPG Biotech & Pharmaceutical Consulting Matthew B. Klein, MD, MS, FACSDavid and Nancy Auth Washington Research Foundation Chair for Restorative Burn Surgery, Associate Professor of Surgery and Adjunct Associate Professor of EpidemiologyUniversity of Washington Randi Kronthal-SaccoAdvisor, CommercializationEdison Pharmaceuticals William D. Shrader, PhDSenior Consultant, InnovationAmpere Life Sciences Peter Barton Hutt, JDSenior Regulatory CounselCovington & Burling, LLP Richard F. Kingham, JDRegulatory CounselCovington & Burling, LLP Gladys H. Monroy, JD, PhDPatent CounselMorrison & Foerster, LLP Michael J. O’DonnellCorporate CounselMorrison & Foerster, LLP


1 Principal Investigators


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