One Shot to Live, New Registry Questions, and More!

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

We’re excited to share several important updates with you this month — including the official launch of our One Shot to Live campaign, a united effort to raise $2.5 million and help advance a clinical trial for SURF1 Leigh syndrome.

We’re also highlighting important new additions to the Leigh syndrome patient registry, including questions focused on treatment priorities and clinical trial perspectives. Every response matters and helps drive research that reflects the needs and priorities of our community.

You’ll also find updates on our new merchandise collection, emerging mitochondrial transplantation research, community fundraising efforts, and upcoming conferences and speaking engagements.

Thank you for being part of this mission and for your continued support of our community.

With gratitude,
The Cure Mito Foundation Team

“Build something that didn’t exist yesterday. Stand up for someone who can’t stand up for themselves. Bring people together who weren’t talking before. That’s leadership. That’s what moves the needle.”

— Harrison Ford —

One Shot to Live Campaign

This month we have officially launched the One Shot to Live — a united campaign to help raise $2.5 million and advance a clinical trial for SURF1 Leigh syndrome.

This is the closest we’ve ever been to a potential lifetime treatment, and we are hopeful that if our community comes together, we can raise the remaining funds needed by the end of the year.

Don't miss our launch video below! View campaign and create your own fundraising page HERE.

One Shot to Live Merch

We teamed up with artist Kaylee Castleberry to create a beach-inspired collection for our SURF1 fundraiser. We love the fun coastal design made to help unite our community around this cause.

All proceeds from the collection go toward accelerating the SURF1 clinical trial and advancing this mission forward.

Check out the full collection HERE.

New Registry Questions: Treatment and Research Priorities

Leigh syndrome patient registry now has new questions focused on understanding patient and caregiver perspectives - particularly around treatment priorities and risk tolerance when considering new therapies or participation in clinical trials.

Your responses will help ensure that research and future trials reflect what matters most to our community. As always, results will be available and shared widely.

First 20 people to enroll or update their profile receive a $25 Visa gift card!

For enrollment instructions, visit our REGISTRY PAGE. If you need help please be in touch with us!

We would like to thank our friends at SDS Alliance and NKH Crusaders for sharing their own similar questionnaires with us, which were incredibly helpful as we developed these new questions. We are also grateful to Dr. Leora Fox, our scientific consultant, and to parents in our community who reviewed and provided valuable feedback to help ensure the questions are meaningful and relevant to families.

New Blog Post: Mitochondria Transplantation for Renewed Energy

Mitochondrial transplantation is an emerging approach focused on restoring energy at the cellular level— by delivering healthy mitochondria into damaged cells.

Cure Mito is partnering with Dr. Jon Brestoff at Washington University in St. Louis to study the potential of this approach for Leigh syndrome and other mitochondrial diseases. Learn more in our most recent blog post.

Thank You for Fundraising

Thank you to the many individuals and families who continue to fundraise in support of Cure Mito. Your dedication, creativity, and commitment are helping to drive research forward and bring hope to our community.

This month we are honored to highlight

"Westin: Built Tough" Fund - Westin was diagnosed with SURF1 Leigh syndrome in 2023, turning his family’s world upside down overnight. Despite the challenges, Westin continues to inspire those around him with his strength, resilience, and joy — fueling hope and the fight for better treatments and a cure. Learn more and donate HERE.

Meet us at upcoming conferences

Kasey will represent Cure Mito Foundation at the International Mito Patients Annual Meeting 2026 on May 30–31 in Angers, France. She will be sharing Cure Mito’s work and discussing opportunities for collaboration across the global mitochondrial disease community.

Sophia will speak at the World Orphan Drug Congress USA 2026 on June 11 on the topic: “Publishing Your Registry Data: A Roadmap for Patient Organizations” — sharing lessons learned from our multiple patient registry publications with the rare disease community.

Our patient registry partner, Sumptuous Data Sciences, will exhibit at Pharma SUG conference in Boston on May 31-June 3 and will have Cure Mito registry and other information available in their booth. Please stop by if you are attending!

Things that might be of interest

Courageous Parents Network will hold a webinar for parents on May 21st:

"Using Social Media: Pros and Perils of Virtual Engagement". Learn more and register HERE.

NIH has developed a new resource for returning research results to the study participants. Please learn more HERE.

Critical Path Institute has a new podcast episode with Ron Bartek, founder of Friedreich's Ataxia Research Alliance (FARA). The episode discusses how collaboration with C-Path helped advance drug development efforts for the Friedreich’s ataxia community. Please listen HERE and learn more about Mitochondrial and Metabolic Disease Taskforce which has the potential to do similar work HERE.

Cure Mito Foundation is a 501(c)(3) nonprofit organization founded by parents and driven by a deep commitment to finding a cure for Leigh syndrome and other mitochondrial diseases. Your tax-deductible donation directly supports research focused on advancing treatments and improving the lives of those affected.

For many ways to give, please click here:

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

Donate Now

You can unsubscribe at any time.

6808 Old Glory Ct
TX 75071
USA