Cultivating the Best Science is Our Best Hope
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The Roadmap to a Cure initiative guides the UMDF mission and focuses on three pillars: Diagnosis, Therapeutic Development and Patient Care. We aim to fast-track science, fund infrastructure and push progress across each pillar.
“UMDF is the largest funder of mitochondrial research outside of the federal government. In the last few years, discovery has streamlined the diagnosis for many and allowed designer therapies to be developed for several rare diseases that may be translatable to mitochondrial diseases. The Roadmap to a Cure provides direction for obtaining a diagnosis, developing care pathways for patients and finding therapies to alleviate symptoms.”
Dr. Bruce H. Cohen
Chair, UMDF Scientific & Medical Advisory Board
DIAGNOSIS
The Challenge
The pathway to a mitochondrial disease diagnosis is not standardized.
Our Commitment
Create a better diagnostic scenario to identify and characterize mitochondrial disease patients based on health information, genetic testing and bio samples.
Our Strategy
- Increasing Awareness
- Improving Diagnoses
- Developing Tools to Measure Mitochondrial Health/Disease
THERAPEUTIC DEVELOPMENT
The Challenge
There is an absence of well-controlled studies within the field and no licensed therapies for mitochondrial disease in the United States.
Our Commitment
Coordinate stakeholders in academia, government and the drug development industry to address validated outcome measures, patient-report outcomes and regulatory guidance to gain treatments more efficiently and quickly.
Our Strategy
- Facilitating Drug Development
- Identifying and Funding Gaps from Basic Science to Clinical Trials
PATIENT CARE
The Challenge
Clinical care for mitochondrial disease patients is often inconsistent, and insurance reimbursement for rare disease care is challenging.
Our Commitment
Leverage the national focus on personalized medicine to develop programs and tools that will advance, optimize and lead to standards of patient care for the mitochondrial disease community.
Our Strategy
- Personalized Medicine
- Patient/Clinical Education
- Developing Coordinated Care Models
- Establishing Centers of Excellence
Clinical Trial Opportunities for Patients
Our best hope for finding treatments and cures is clinical trials. For research studies to be effective, a large amount of data from a large pool of participants is essential. We urge patients to join the fight and engage in clinical trials to help make a difference for future generations.
Stay up-to-date on the latest news and updates on clinical trials. Visit the UMDF Clinical Trials page.
Interested in applying for a UMDF Research Grant?
Annual UMDF Grant Prize Winners
Award Winners at MitoMed 2026
U.S.-based United Mitochondrial Disease Foundation (UMDF) and Australia-based Mito Foundation are proud to announce eight research grants totaling $500,000 USD have been awarded as part of the 2026 Research Grant Program.
2026 Principal Investigator Grants ($100k Awards)
- Dr Jiang-Hui (Sloan) Wang, PhD, Centre for Eye Research Australia
Next-Generation Mitochondrial ND4 Gene Therapy for LHON: Improving Delivery and Mitochondrial Targeting - Mary Herbert, PhD, Monash University
Development of regulatable mitoTALENs to increase the efficacy of mitochondrial replacement therapy - Javier Triñanes-Ramos, PhD, Amsterdam Leukodystrophy Center, Emma Center for Personalized Medicine, Pediatric Neurology, Emma Children Hospital, Amsterdam University Medical Center
Gene therapy in humanized Dars2 mouse model that recapitulates LBSL neuropathology
2026 Postdoctoral Fellow Grants ($50k USD Awards)
- Chiara Guarona, PhD, Fondazione IRCCS Istituto Neurologico Carlo Besta
Early Pharmacological Interention in SURF-Related Leigh Syndrome Using Sildenafil - Alvaro J. Narbona-Perez, PhD, University of Utah
Function and regulation of the mitochondrial disease-causing metabolite transporter SLC25A19 - Melis Kose, MD, PhD, Children’s Hospital of Philadelphia
OPERA (OPA1-Related Energetics Rescue): Translational Validation of 20 Therapeutic Candidates in OPA1 Patient-Derived Retinal Ganglion Cells
2026 Graduate Student Grants ($25k USD Awards)
- Nissa Carrodus, BSc, The University of Queensland
mRNA-induced protein replacement therapy for mitochondrial DNA depletion syndromes - Lily Farmerie, BS, University of Pittsburgh
A precision medicine approach targeting cardiovascular manifestations of m.3243A>G borne primary mitochondrial disease
Your Dollars at Work
Your donations power our ability to support science dedicated to mitochondrial disease research.
million in grants awarded
million stimulated in government grant follow on funding
labs funded and launched
million dedicated to Leigh Syndrome Roadmap Initiative
Making an Impact in Drug Development
UMDF recognizes industry as an essential partner in developing treatments and cures for mitochondrial disease.
Industry Advisory Council
Facilitating Drug Development
There is a need to generate more urgency within the drug industry to invest and develop therapeutic treatments focused on mitochondrial disease.
No single organization can take on mitochondrial disease alone. UMDF has gathered the leading mitochondrial disease patient advocacy groups from around the globe to form and fund The Leigh Syndrome International Consortium. This Roadmap to a Cure project showcases our active dedication to find the best science wherever it is located in the world.
UMDF interacts with multiple organizations and is the nucleus of many infrastructure projects dedicated to mitochondrial disease clinical research and patient care. UMDF is collaborating with key stakeholders to create a single hub essential for sharing and dispersing critical information to benefit the entire mitochondrial disease community.
UMDF stewards mitoSHARE, a worldwide patient-populated registry initiative.
UMDF is an executive committee member of the North American Mitochondrial Disease Consortium, a clinical research network funded by NIH.
UMDF plays an active role and co-funds the Mitochondrial Care Network to optimize clinical care.
UMDF collaborates with the Mitochondrial Disease Sequence Data Resource Consortium, a partnership with Children’s Hospital of Philadelphia to consolidate research data.