Table 1 Speakers at the Leigh Syndrome Symposium

Kasey Woleben

Cure Mito Foundation

Introduction

Vamsi K. Mootha, MD

Harvard Medical School, Broad Institute of Harvard and MIT

Keynote: ​Advances in Leigh Syndrome Research: Sterling Past, Golden Future

Saima Kayani, MD

University of Texas Southwestern

Medical Center

Leigh Syndrome 101

Steven J. Gray, PhD

University of Texas Southwestern

Medical Center

General Overview of Nervous System Targeted Gene Therapy

Michal Minczuk, PhD

University of Cambridge, Pretzel Therapeutics

Mitochondrial Genome Engineering In Vivo

Jessica L. Fetterman, PhD

iCAMP lab, Boston University School of Medicine

Gaining Insights into Mitochondrial Diseases Using Stem Cells

Alessandro Prigione, MD, PhD

University of Düsseldorf, Germany

Patient-Derived Stem Cells and Brain Organoids for Modeling and Drug Discovery of Leigh Syndrome

Simon C. Johnson, PhD

University of Washington, Seattle Children’s Research Institute

Immune-Mediated Disease Pathogenesis in Leigh Syndrome

Joseph J. Bellucci, PhD

Rarebase

Applying A Precision Medicine Drug Discovery Platform to SURF1 Leigh Syndrome

Anne N. Murphy, PhD

Cytokinetics

Challenges in Drug Discovery for Mitochondrial Disorders

Matthew B. Klein, MD, MS, FACS

PTC Therapeutics

Insights from over a Decade of Experience in Mitochondrial Disease Drug Development

Volkmar Weissig, ScD, PhD

Midwestern University

Development of New Drugs for Mitochondrial Diseases with a Focus on Leigh Syndrome

Sophia Zilber

Cure Mito Foundation

Leigh Syndrome Global Patient Registry Updates

Rhonda Facile, MS

Partnerships and Development, CDISC

CDISC and How Data Standards Can Help Drive Development of Mito Treatments

Parag Shiralkar, MS, MBA

Sumptuous Data Sciences

Interoperability of Leigh Syndrome Patient Registry Data with Regulatory Submission Standards

Alexandre Bétourné, PhD, PharmD, PMP

Critical Path Institute

The Rare Diseases Cures Accelerator Data and Analytics Platform: Accelerating Drug Development for Rare Mitochondrial Disorders.

Shannon Rego O’Rourke, MS, CGC

Allstripes

SURF1-related Leigh Syndrome Health Data Insights

Parents of Patients with Leigh Syndrome

N/A

Parent panel

Jennifer Linnebach (USA)

Krisztina Ferencz (Romania)

Jorgelina Alvarez Barral (Argentina)

Rachel Kramer, PhD

Clinical Psychologist, Private Practice

Parenting Children with Mitochondrial Disease: Finding Space for Parental Well-Being

Jessica L. Fetterman, PhD and Kasey Woleben

iCAMP lab, Boston University School of Medicine and Cure Mito Foundation

Closing Remarks