Year-End Reflections, New Research, Families, and More !

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

As we reflect on the year coming to a close, we’re grateful for how much we’ve accomplished together. This year, our team continued to grow, we hosted our 4th Annual Leigh Syndrome Symposium, published a new paper capturing our journey and the latest results from the Leigh Syndrome Global Patient Registry, launched regular family meetups, shared the stories of families across our community on our website, and awarded more than $400,000 to support Leigh syndrome research.

We share these milestones with gratitude and reflection, while also acknowledging another loss in our community — a reminder of the urgency and heart behind our mission.

In this spirit, we’re also sharing updates on our new publication, a successful Giving Tuesday, new family-focused initiatives, and more.

Looking ahead to the coming year, we look forward to launching a patient priorities survey, sharing detailed updates on the research we fund, and convening a research retreat and workshop — with more to come.

You’ll find all the details in the sections below. Thank you for being part of our mission and for standing with our community every step of the way.

With gratitude,
The Cure Mito Foundation Team

“ We are the ones we’ve been waiting for. ”

— June Jordan —

Our paper is published!

We’re excited to share that our new paper, Expanding Research and Care for Leigh Syndrome: Efforts of a Patient-Led Advocacy Organization, is now published in the Journal of Research Involvement and Engagement.

This paper highlights our growth as a patient-led organization — from the Leigh Syndrome Global Patient Registry to expanding research and data-sharing efforts — all made possible by our incredible families and partners.

Read the full paper HERE.

Leigh Syndrome Global Patient Registry Update

One of the things you'll find in our new paper are updated results from the Leigh Syndrome Global Patient Registry, showing incredible growth since our 2023 publication.

Thanks to families, clinicians, and researchers worldwide, this expanding data is helping drive research, natural history insights, and progress toward better outcomes.

If your loved one has Leigh syndrome - or has passed away from it - please join the registry.

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

Learn More

Giving Tuesday: Thank You for Showing Up

Because of you, we met our Giving Tuesday goal and unlocked the full $10,000 match. Every donation, share, and message of support made this possible — and we are deeply grateful for this community.

Our Giving Tuesday fundraiser remains open through the end of the year. You can still support families and fund Leigh syndrome research by donating HERE. 💚

Medical mom gifts guide

Inspired by our new Powerhouse Parents family meetups, we’re sharing a Medical Parent Gift Guide, created by families, for families.

This initiative is led by Emily Andersen, our new Family and Education Director and mom to Jack, who has Leigh syndrome.

Explore the guide HERE.

You can also find these and more in our Amazon StoreFront.

We Remember Millie

It is with heavy hearts that we share that this month we lost Millie Jones to Leigh syndrome.

Millie’s family has been a valued Cure Mito partner family, raising awareness and funds for research, and Millie brought so much joy to her family every single day. She will always be remembered for the love she inspired and the light she brought into the lives of those who knew her.

We hold Millie’s family close in our hearts and honor her memory.

FDA Grants Type A Meeting to Saol Therapeutics

The FDA granted a Type A meeting on December 18th to review new and expanded datasets and to help identify a feasible path forward for SL1009 in Pyruvate Dehydrogenase Complex Deficiency (PDCD)i thout the need for an additional clinical trial.

Learn more in the PRESS RELEASE.

Additionally, the Hope for PDCD Foundation delivered a petition to the FDA urging approval of the first targeted PDCD treatment.

Recent Paper Highlights

This month, we’re sharing two important papers that shed light on how families experience genomic testing results and the ongoing emotional and communication needs that may persist long after testing.

Fathers’ and Mothers’ support needs and support experiences after rapid genome sequencing

A new study in the European Journal of Human Genetics highlights that emotional well-being, anxiety, depression, and unmet support needs can persist for years after rapid genomic sequencing—especially for families whose child receives a genetic diagnosis. The findings underscore the importance of sustained emotional and community support alongside advances in genomic medicine.
Read the full paper HERE.

Challenges associated with disclosing results from whole genome sequencing to diagnose paediatric rare diseases: analysis of parent–clinician interactions

A new study in the Journal of Medical Genetics examines how families experience the return of whole genome sequencing results for children with rare neurological or developmental disorders. Through analysis of parent–clinician consultations, the authors highlight key communication challenges around diagnosis, follow-up, unexpected findings, and uncertain prognoses as genomic testing becomes more integrated into rare disease care.
Read the 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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