Giving Tuesday, Family Stories, PDCD Petition and More!

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

We’re excited to share our November updates with you. This month, we highlight our upcoming Giving Tuesday campaign, new family stories from our Community Engagement Director, Lauren Escobedo, an important petition supporting PDCD families, a reminder to join or update the Leigh Syndrome Global Patient Registry, recent FDA news impacting rare disease drug development, and more!

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

“ There are two ways of meeting difficulties: you alter the difficulties, or you alter yourself to meet them.”

— Phyllis Bottome —

Giving Tuesday is on December 2, 2025

Join us in supporting Leigh syndrome research focused on gene therapy, mitochondrial replacement therapy, and drug repurposing.

Thanks to a generous match, all donations will be doubled up to $10,000 — let’s DOUBLE our impact for children and families affected by Leigh syndrome!

Please DONATE TODAY!

Your Journey can Inspire Others!

Since September, our Community Engagement Director, Lauren Escobedo, has been sitting down with families to listen, learn, and help share their powerful stories.

Each story is a reminder of the strength, hope, and resilience within our community. You can check out all the beautiful stories HERE.

Would you like to share your family’s journey? Lauren would love to connect with you. Please reach out to her at lauren.escobedo@curemito.org

Support PDCD families!

Sign and share the petition today to support PDCD families. We’re asking the FDA to consider DCA — a treatment that many families report has helped stabilize their children — and to engage directly with the PDCD community to understand their experiences.

This isn’t about lowering scientific standards; it’s about applying the regulatory flexibility that rare diseases require and ensuring that meaningful patient outcomes are part of the decision-making process.

Sign the petition HERE.

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

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.

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

Learn More

New FDA “Plausible Mechanism” Pathway

The FDA has introduced a groundbreaking approval pathway that could transform treatment development for ultra-rare genetic diseases. Instead of requiring traditional clinical trials—often impossible for rare diseases—this new approach allows therapies to advance based on a well-understood biological mechanism.

The pathway outlines five key elements: a clearly defined molecular cause, a therapy designed to directly correct that defect, strong natural history data, evidence that the treatment engages its target, and meaningful clinical benefit—even in a single patient.

Learn more in a paper published in the New England Journal of Medicine.

Recent Paper Highlights

Engaging patient-led rare disease organizations to advance research – through the lens of Bardet–Biedl syndrome

A new article in the Journal of Cell Science by Timothy Ogden, Bendert de Graaf, and Tonia Hymers explores how meaningful partnerships between researchers and patient-led rare disease organizations can advance science and improve outcomes. Using Bardet–Biedl syndrome as an example, the authors highlight the importance of respectful listening, shared priorities, and mutual trust.
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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