The Empower & Inspire Symposium is the only global virtual event dedicated to Leigh syndrome and is widely attended by the entire mitochondrial disease community.
It unites patient families, industry professionals, academic researchers, and clinicians, providing a unique platform to share information and insights that advance research and empower families.
Join us on September 16, 2025 to explore advances in care, research, and collaboration. Agenda is coming soon!
Executive Director, President and Co-Founder, Cure Mito
Parent
A personal journey through parenting a child with a rare disease, evolving technology, and the daily choices that blend clinical care with a mother’s instinct. This talk shares how one family balances science and soul, navigating advanced interventions with intuition and hope for the future.
Assistant Professor, Umass Chan Medical School
Dr. Ling will present the design and preliminary data on a novel gene therapy strategy developed for patients with MTATP6 m.8993T>G mutation.
Professor, UT Southwestern Medical Center
Dr. Gray will discuss the latest developments in the effort to develop a gene therapy treatment for SURF1-related Leigh Syndrome. This will be followed by an overview of the steps to take a lead candidate through IND-enabling studies and into a first-in-human clinical trial.
Associate Professor, Washington University School of Medicine in St. Louis
Mitochondria transplantation is novel therapeutic procedure in which isolated mitochondria are administered with therapeutic intent. In this talk, Dr. Brestoff will describe our efforts to develop this technology to treat Leigh Syndrome.
Professor of Pediatric Metabolic Medicine, University of Düsseldorf
Dr. Prigione will discuss recent progress in understanding and treating Leigh syndrome with iPSCs and brain organoids, including innovative therapies discovered using deep learning. This work led to an EMA Orphan Drug designation for a repurposable compound and a clinical trial now in development. Collaborations with clinicians aim to bring these findings to patients with pediatric mitochondrial diseases.
Professor, University of Cambridge
TBD
Assistant Professor of Genetics and Pediatrics, Icahn School of Medicine at Mount Sinai
Dr. Elsharkawi will discuss nutrition and mitochondrial disease, and new insights in recent years about links between the gut microbiome and the mitochondria.
Associate Professor and Investigator, Gladstone Institutes and UCSF
Both oxygen deprivation (hypoxia) and excess (hyperoxia) are toxic, contributing to major diseases such as heart attack, stroke, and lung injury. While their molecular mechanisms remain unclear, our recent work uncovers how hyperoxia destabilizes specific iron-containing protein complexes, driving cellular damage. These findings reveal a novel therapeutic approach that we aim to extend to inborn errors of metabolism, aging, and other metabolic disorders.
Co-founder and CEO, Unravel Biosciences
Unravel Biosciences partnered with Cure Mito and over 20 families to analyze 4 different Leigh syndrome-causing genes. Our results show drug candidates that may benefit multiple Leigh syndrome patient groups, independent of the causative gene.
CEO, Perlara
Dr. Perlstein will share preclinical and clinical summaries of the past 5 years of using patient avatars to repurpose drugs for mitochondrial diseases like Leigh syndrome.
Executive Director, C-Path
The presentation will provide an overview of the Rare Mitochondrial Diseases Task Force mission, with an update on near term deliverables including the development of recommendations to enhance registry data collections, and discuss long-term goals with cases examples of drug development tools that our group aim to generate.
Head of Medical Affairs, Saol Therapeutics
This talk will introduce an innovative model for rare disease clinical trials designed to streamline the drug development process and accelerate patient access to treatment. By integrating adaptive trial designs, regulatory strategy optimization, this model aims to overcome traditional barriers in rare disease research. The goal is to show how a more efficient, patient-centered approach can expedite clinical evaluation while maintaining scientific rigor and regulatory compliance.
Head of Medical Affairs, Saol Therapeutics
Head of Market Access, Saol Therapeutics
In this talk Saol Therapeutics team will discuss the clinical trial results of SL 1009 for the treatment of PDCD.
The symposium is moderated by Dr. Leora Fox, Fox Scientific Consulting
Executive Director, President and Co-Founder, Cure Mito
Professor of Pediatric Metabolic Medicine, University of Düsseldorf
Assistant Professor of Genetics and Pediatrics, Icahn School of Medicine at Mount Sinai
Head of Clinical Operations, Elpida Therapeutics
Associate Professor and Investigator, Gladstone Institutes and UCSF
Co-founder and CEO, Unravel Biosciences
Assistant Professor, Umass Chan Medical School
Professor, University of Cambridge
Parent
Professor, UT Southwestern Medical Center
Head of Market Access, Saol Therapeutics
Head of Medical Affairs, Saol Therapeutics
CEO, Perlara
Executive Director, C-Path
Associate Professor, Washington University School of Medicine in St. Louis
Owner, Fox Scientific Consulting, LLC
This conference is also made possible in part by a grant from the Chan Zuckerberg Initiative DAF, an advised fund of the Silicon Valley Community Foundation.
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