Research Retreat Deadline, Registry News, Community Gathering, and More!

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

As October comes to a close, we’re excited to share several key updates. The deadline to submit proposals for the Leigh Syndrome Research Retreat and Workshop is approaching—don’t miss this opportunity to collaborate and advance the field.

We’re also highlighting milestones from the Leigh Syndrome Global Patient Registry and reminding families to join and contribute to this vital effort.

Families and caregivers are invited to an informal virtual gathering with our Community Engagement Director, Lauren Escobedo, on October 31—a chance to connect, share, and learn more about our community.

You’ll also find a few recent research highlights and news updates below.

Thank you for being part of our mission—your support and engagement continue to drive progress for everyone affected by Leigh syndrome.

With gratitude,
The Cure Mito Foundation Team

“No one has ever achieved anything from the smallest to the greatest unless the dream was dreamed first.”

— Laura Ingalls Wilder

Request for Proposals: Leigh Syndrome Research Retreat and Workshop - Deadline is November 1st

Just a few days left! Researchers and medical professionals - submit your proposals to join our two-day, in-person retreat and workshop focused on advancing research and treatment for Leigh syndrome. Learn more and apply.

Submission Deadline: November 1, 2025

Are you a part of the 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.

Our Achievements Since 2021

420+ participants enrolled from 49 countries
2 peer-reviewed papers published; a third under review
5 scientific posters presented
Data interoperable with both CDISC and OMOP — the only patient-driven mitochondrial disease registry to have achieved this capability!
Transparent data practices: openly sharing how we analyze data, ensure data quality, and enable responsible data sharing
Registry data can connect with other data sources through a CRID (Clinical Research ID)

Families: Learn more about how to register.
Researchers and professionals: Contact us to explore how the registry can support your work.

Learn More

Join us for a gathering and conversation with our Community Engagement Director, Lauren Escobedo!

Parents, caregivers, and families are all welcome to join an informal discussion and time for connection on October 31 at 11 a.m. CT / 12 p.m. ET.

Join via Zoom: https://us02web.zoom.us/j/81347922435?pwd=W9tPbw35r1Illo9vaXhDByQEbZwnMm.1

Congratulations to C-Path!

Congratulations to C-Path on receiving the 2025 Innovation Award from the Reagan-Udall Foundation for the FDA, recognizing groundbreaking advances in regulatory science that measurably improve public health.

Founded in 2005 in response to the FDA’s Critical Path Initiative, C-Path builds neutral, global collaborations that deliver regulatory-grade tools, data, and methods used across diseases and modalities. Learn more about C-Path and the award HERE.

We are proud to collaborate with C-Path through the Mitochondrial Diseases Task Force, bringing together patient organizations, researchers, regulators, and industry partners to accelerate development of therapies for mitochondrial diseases. Learn more about the taskforce HERE.

Update: Saol Therapeutics & Pyruvate Dehydrogenase Complex Deficiency (PDCD) Treatment

Saol Therapeutics has received a Complete Response Letter (CRL) from the U.S. Food & Drug Administration regarding its investigational drug SL1009 (sodium dichloroacetate, DCA) for the treatment of PDCD.

Saol is working with the FDA to determine next steps. Access to SL1009 will continue through current clinical trials and expanded access programs. The company remains dedicated to helping patients and families affected by this rare mitochondrial disease.

You can learn more through the following links:

Saol press release

Letter signed by over 80 of the leading researchers and clinicians in the mitochondrial disease space

Recent Paper Highlights

Expert Reactions to Mitochondrial Donation & Embryo Testing Research

We invite you to read the expert commentary on two recent papers published in The New England Journal of Medicine, covering mitochondrial donation and preimplantation genetic testing for mitochondrial disease. The insights shared by leading scientists explore the current implications, challenges, and future directions of this evolving field.
Read the full expert reactions HERE.

Outcomes misaligned in mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS): implications for trial design

A new study in Brain Communications by Stefanetti et al. reports that in MELAS, patient-reported outcomes don’t always match objective measures - highlighting the need for multi-dimensional outcomes in mitochondrial disease trials. Read the full paper HERE.

Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK

A new study by Peay H et al. surveyed 263 patients and caregivers in the U.S., U.K., and other countries to assess the maximum acceptable risk of death (MAR) from non-curative gene therapy for Duchenne muscular dystrophy. Risk tolerance increased with advancing disease, ranging from 2.4 % in newborns to 5.4 % in later stages, with some participants accepting risks above 10 %. Read full paper 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

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