Leigh Symposium Registration is Open, 2 Grants Awarded, and more News!

Dear Friends, Partners, and Supporters of the Cure Mito Foundation,

Welcome to our May 2025 newsletter!

We’re excited to share several important updates with you this month:

Registration is now open for our 4th Leigh Syndrome Symposium—don’t miss the chance to join us. Register today!
We’ve awarded two new research grants to advance Leigh syndrome and mitochondrial disease research.
We’re also highlighting recent events, community news, and ways to stay engaged.

Thank you for being part of this growing and dedicated community.

With gratitude,
The Cure Mito Foundation Team

"Do not wait for someone else to come and speak for you. It's you who can change the world"

- Malala Yousafzai -

Registration is Open!

Empower and Inspire: 4th Annual Virtual Leigh Syndrome Symposium

The Empower & Inspire Symposium is the only global virtual event dedicated to Leigh syndrome and is widely attended by the entire mitochondrial disease community.

Please learn more and register here.

If you are interested in being a sponsor please be in touch with us!

We’re proud to announce two new research grants supporting innovative mitochondrial disease research!

🔬 $202,000 to Dr. Steven Gray at UT Southwestern Medical Center to advance the development of SURF1 gene replacement therapy.

🧬 $34,000 to Dr. Ibrahim Elsharkawi at Icahn School of Medicine at Mount Sinai for his study, “Investigating the Role of the Gut Microbiome in Primary Mitochondrial Disease.”

More updates on these will be shared at the Leigh syndrome symposium on September 26th, don't miss it!

Thank you Dr. Dr. Qinglan Ling and Dr. Sarah Holbrook for representing us at the 8th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)!

Dr. Qinglan Ling presented a talk titled “Achieving ‘Just Right’: Preclinical Development of a Gene Therapy for SURF1 Leigh Syndrome Using Two Disease Models” on Tuesday, May 13th.

Dr. Sarah Holbrook, PhD presented a poster titled “Investigating allotropic expression and mitochondria-targeted base editors as therapeutic strategies to alleviate MT-ATP6-associated Leigh syndrome.”

We can't thank both of them enough for their hard work and dedication to our community!

Join Leigh Syndrome Patient Registry

If your loved one has Leigh syndrome - or has passed away from it - please consider joining the world’s largest Leigh Syndrome Global Registry.

All findings are shared back with the community - because your experiences matter.

Learn More

Important registry reminders for patients:

1) Please obtain your Clinical Research ID (CRID) by visiting www.thecrid.org. You will be asked to enter your CRID in the last question on the registry survey.

2) When you enroll in the registry, please ensure you complete both surveys. Without this, we cannot utilize or learn from your data.

2) Please remember to enter genetic mutation whenever available and, if possible, upload the corresponding genetic report.

3) You can enroll or update your registry information HERE. If you are having trouble logging in, please reach out to cords@sanfordhealth.org.

We attended the Mito Transfer conference!

Kasey attended the Mitochondrial Transplantation and Next Generation Therapeutics Conference at at Hofstra University in Long Island, NY — thank you to the organizers for creating space to collaborate, share, and learn together.
📸 Pictured here: Kasey Dr. Becker Lance

We participated in the Primary Mitochondrial Diseases Workshop

We are honored to have had a chance to participate in a Primary Mitochondrial Diseases Workshop organized by the Reagan-Udall Foundation for the FDA. Recordings to come soon!

Papers and Resources Highlights

Congratulations to Dr. Alessandro Prigione and team on identifying sildenafil as a promising candidate for Leigh syndrome through a large drug repurposing screen using patient-derived neural cells. In lab models, sildenafil restored neurodevelopmental pathways, improved brain organoid function, and extended lifespan in mice. Compassionate use in six patients with MT-ATP6 mutations also showed improvements in motor function and quality of life. We are proud to have contributed to this important research.

Pluripotent stem cell-based drug discovery uncovers sildenafil as a treatment for mitochondrial disease | medRxiv

New study by Nijim et al. (JAMA Netw Open, 2025) highlights key factors behind successful rare disease drug repurposing efforts, offering a roadmap for nonprofits, collaborators, and policymakers to drive impact through systematic, data-driven approaches. Rare Disease Drug Repurposing | Health Policy | JAMA Network Open | JAMA Network

Serious Illness Conversation Guide, developed by Ariadne Labs, helps clinicians talk with patients about what matters most — their values, goals, and understanding of their illness.

Serious-Illness-Conversation-Guide.2023-05-18.pdf

Find more papers that maybe of interest to our community HERE.

Leigh Syndrome Families Checklist

Join the Global Leigh Syndrome Patient Registry

Add your doctors to the medical provider directory

Join our Facebook group

Become a partner family

Volunteer

Email info@curemito.org with your skills and time availability

Join annual Leigh syndrome symposium

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Cure Mito Foundation is a 501(c)(3) nonprofit organization led by parents who volunteer their time to search for a cure. 100% of your donations are tax-deductible and will go directly to research dedicated to mitochondrial diseases.

For many ways to give, please click here:

https://www.curemito.org/ways-to-give

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