New Research Opportunities, Empower & Inspire Symposium Updates, and More Community News ⭐

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

We’re excited to share several important updates with you this month, including new opportunities to participate in Leigh syndrome research, the newly released agenda for our 5th Annual Empower & Inspire: Leigh Syndrome Symposium, and upcoming Mito Awareness Week activities.

We’re also highlighting updates from the Leigh Syndrome Global Patient Registry, an important PDCD clinical trial update with a call to action for the community, highlights from our incredible family fundraisers and more.

Thank you for being part of this mission and for your continued support of the Leigh syndrome community.

With gratitude,

The Cure Mito Foundation Team

“People are capable, at any time in their lives, of doing what they dream of. ”

— Paulo Coelho —

Empower and Inspire: 5th Annual Leigh Syndrome Symposium

The agenda for the 5th Annual Empower & Inspire: Leigh Syndrome Symposium is now live!

Please find the full agenda HERE.

Registration will open in August, please follow us and stay tuned for updates!

More to come during Mito awareness week:

September 17th is a Leigh Syndrome Awareness Day! Learn more about this day and how to participate HERE.

Color for Mito contest and prizes are coming. Stay tuned for more info!

We have several research studies for you to participate in. Please learn more below!

Developing a gene therapy for MT-ATP6 Leigh syndrome patients using patient derived cells with Dr. Qinglan Ling

We are currently seeking 3 individuals with each of the following genetic variants - m.8993T>C, m.8993T>G, m.9176T>G, and m.9176T>C - as well as 3 healthy controls for each variant to participate.

Gut microbiome study

We're seeking volunteers for our gut microbiome studies at Icahn School of Medicine. If your child ages 1+ with SURF1, ECHS1 or MT-ATP6 Leigh syndrome can participate, we need you!
Study involves sharing medical records, optional diet logging, and sending stool samples (we cover shipping).

Ideal healthy volunteers are close in age to the affected child. Siblings or parents can join too.

MitoMarkers Project for SURF1 Leigh Syndrome

This study is being conducted by Dr. Rohit Sharma and Dr. Melissa Walker at Massachusetts General Hospital. It will take place in Dallas on Saturday, September 26, 2026.

The study aims to collect blood, urine, and dried blood spot samples from 10 individuals with SURF1 Leigh syndrome, along with healthy controls, to investigate potential biomarkers.

If you are interested in participating or would like more information about the studies above, please email kasey@curemito.org.

Leigh syndrome global patient registry

Every person with Leigh syndrome counts. If you or a family member has been diagnosed with Leigh syndrome - or has passed away from the disease - please join the Leigh Syndrome Global Patient Registry.

Registry results are always shared with the community and made available on our website.

If you're already enrolled, we recently added 10 new questions about treatment priorities and clinical trial participation, and we would be grateful if you could take a few minutes to update your information.

Registry links that may help:

Find all information about the registry HERE.

Use AI assistant to help you enroll and complete the registry questions.

Browse registry published papers HERE.

View high level data overview HERE.

Article about the registry in CNBC Cures Newsletter.

Researchers can connect their own research data to participant's data in the registry via a CRID.

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

💚 Aadya's friends and family for the $33,000 donation towards Aadya's One Shot to Live campaign.

💚 AJ and her family for raising awareness and funds for Leigh syndrome during the Fourth of July parade!

💚 An anonymous donor who gave $25,000 donation towards AJ's campaign bringing her campaign total to over $68,000 for SURF1 gene therapy!

💚 Delta and her family and friends for hosting a lemonade stand to benefit Cure Mito!

💚 Zander and his family for raising funds through a lottery ticket fundraiser.

PDCD Trial Update & Call to Action

Saol Therapeutics has shared the New Drug Application for DCA to treat PDCD has officially been resubmitted to the FDA with additional analyses and data.

Please read the full letter from Saol Therapeutics to the community HERE.

The review could take as long as six months, but with the process already approaching 600 days, we need your help to shorten that timeframe. PDCD families don’t have time to wait. Use THIS LINK to encourage your House member and Senators to ask the FDA to prioritize a timely DCA review.

New Preprint Highlights Registry Harmonization Effort

The mitochondrial disease community has many patient registries, but differences in how data are collected can make it challenging to combine information across studies.

We're proud to have spearheaded the creation of C-Path's Primary Mitochondrial Diseases Task Force and contributed subject matter expertise to the manual harmonization of four mitochondrial disease registry data dictionaries: the Cure Mito Foundation Leigh Syndrome Global Patient Registry, Cure ARS, Hope for PDCD Foundation, and The Champ Foundation.

Learn more in the new preprint.

Cure Mito Resources

Did you know that Cure Mito offers a variety of resources for individuals, families, and healthcare professionals? Explore some of them below.

Meet Leigh Syndrome Heroes - family stories

Support for families and newly diagnosed care packages

Information about family planning for Leigh syndrome and mitochondrial disease

Resources for Medical Providers

Plain language glossary of research and clinical terms

Papers that might of interest to our community

Amazon store front

Things that might be of interest

We enjoy staying up to date on developments that may be of interest to our community and sharing them with you.

Defining kindness in healthcare: Perspectives from international experts - ScienceDirect

Exploring the principles of logotherapy in genetic counseling: Enhancing decision‐making, adaptation, and justice - Chanouha - 2026 - Journal of Genetic Counseling - Wiley Online Library

What Clinicians Mean When They Say, “The Parent Is Always Right”: A Reflection - Courageous Parents Network

NIH's All of Us Research Program is now the largest integrated genomics and health database in the world | National Institutes of Health (NIH)

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