New Registry Dashboard, Research & Community Highlights

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

We’re excited to share our April updates with you. This month, we highlight a new blog exploring the connection between mitochondrial disease and the gut microbiome, the launch of our patient registry data dashboard, and continued efforts to elevate the voice of our community through advocacy and research.

We’re also proud to share a meaningful new children’s book, recognize the impact of our incredible fundraising community, and spotlight a family working urgently to advance gene therapy research.

You’ll find all the details in the sections below. Thank you for being part of our mission and for your continued support.

With gratitude,
The Cure Mito Foundation Team

“ The most effective way to do it is to do it.”

— Amelia Earhart —

New Blog Post: Mitochondrial Disease and the Gut Microbiome

What does the gut microbiome have to do with Leigh syndrome?

From mitochondrial function to inflammation and diet, our scientific collaborator Leora Fox explains why this emerging area of research matters.

Learn more HERE.

New: Patient Registry Data Dashboard

We’re excited to share our new registry dashboard, offering a high-level snapshot of registry data—including demographics, diagnosis, and genetic - designed to make information from our community more accessible and transparent.

Please note that this is not a live view—the current snapshot represents data as of March 2026. As the registry continues to grow and evolve, the dashboard will be updated regularly to reflect the latest insights.

You can view the dashboard HERE.

Coming soon to the patient registry: Treatment and Research Priorities

In the coming weeks, registry will have 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.

These updates will help ensure that research and future trials reflect what matters most to our community.

Please follow us to learn when these questions go live!

Please visit our website where you can learn more or join the registry.

Learn More

New research:

Accelerating Leigh syndrome drug discovery through deep learning screening in brain organoids

Excited to share new research led by Dr. Alessandro Prigione and team, which we’re proud to have supported and co-authored, exploring a novel approach to accelerate drug discovery in Leigh syndrome using brain organoids and computational screening.

The study identifies azole compounds, including talarozole and sertaconazole, as potential repurposable treatments. Please read the full paper HERE.

RARE Revolution Feature: Our Top Three Unmet Needs & Key Priorities with Industry

We’re grateful to have shared our top three unmet needs and key priorities with industry in RARE Revolution Magazine®, helping bring the voice of the Leigh syndrome community to a broader audience.

We shared the following key asks:

Include pediatric patients in clinical trials
Share data through neutral platforms such as C-Path
Engage with patient groups authentically and thoughtfully

You can read the article HERE.

New Children's Book: The Tiny City Inside Us

A beautiful new children’s book, The Tiny City Inside Us: A Story About Leigh Syndrome, was written by Desiree L Vennerholm in memory of her daughter, Zoë. 💛

Please read more about

Zoë and her story HERE.

You can order the book HERE. All proceeds from the book are donated to support the work of Cure Mito Foundation.

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

"Help Cure Ruby And Campbell" Fund - Ruby and Campbell are siblings, both living with SURF1 Leigh syndrome. Learning that both of their children have a terminal disease with no clear prognosis or life expectancy has been devastating for their family, who face the reality of anticipatory grief each day. Yet, Ruby and Campbell are currently doing well and continue to bring joy and light to those around them. Learn more and donate HERE.

Scotland to the USA: a family fundraiser for Cure Mito - The Dryburgh, Dodwell-Hill and Barratt families are uniting to run/ walk/ hobble 60 miles over 2 days on the Isle of Arran in Scotland in the honor of Delta who is living with SURF1 Leigh syndrome. Learn more an donate HERE.

We joined IMP

We would like to share with the community that Cure Mito is now a member of IMP (international mito patients). We look forward to this collaboration and will keep the community informed on the progress and outcomes it brings.

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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