Listen to a summary sharing the key updates shaping the mito community right now.
The future of mitochondrial disease (mito) is shifting in ways we once only hoped for. When Mito Foundation began nearly 16 years ago, mito was considered a group of rare, hard-to-diagnose and untreatable conditions. Today, we're seeing real change.
In the past few months, we've seen the U.S. Food and Drug Administration approve 2 new medicines for Barth Syndrome and Thymidine Kinase 2 deficiency (TK2d). It's a moment many families around the world have waited decades for. More than 60 potential treatments are now in development, with over 20 already in clinical studies. It's an extraordinary step forward.
Here in Australia, Mito Foundation's role is to make sure decisions about access and funding fully recognise the lived experience of people with mito. We're committed to working alongside you, and with our partners, to make sure new opportunities reach the Australian community. Read more about these approvals and what they mean for you.
We remain committed to finding treatments, and one day a cure, for everyone impacted by mito. You can help drive progress by donating to our Christmas appeal. Each gift will be tripled up to 24 December thanks to a generous anonymous donor (some conditions apply). We're grateful for your support and for sharing the appeal with your networks.
We're also celebrating the grant of the first licence for the Australian mitochondrial donation pilot program. While it's taken longer than expected, and we know delays have real impacts, this milestone brings us closer to a new reproductive option that could profoundly change lives for the better.
Sean Murray
CEO, Mito Foundation
Mito Community Summit recordings now available
You can now access:
- Video recordings of all expert sessions
- Audio versions for on-the-go listening
- Written summaries of key points
A snapshot of what we're working on
The RISE Project:Â improving support for people with rare, progressive conditions
People living with rare, progressive diseases often struggle to find the emotional, practical and disability supports they need — especially as their condition changes.
The RISE Project (Rare and progressive disability: Inclusive support and empowerment) aims to change this.
The RISE Project is:
- creating simple, accessible resources to help people find emotional, practical and disability supports.
- highlighting how supports need to change as conditions worsen and disability impacts change.
It builds on lived experiences shared through the Mito Stories Project, including challenges navigating services and accessing appropriate care. We’re also working alongside other rare disease organisations to learn from their communities.
This work is possible thanks to a Rare Voices Australia (RVA) Partner Project Grant as part of the Rare Disease Disability Project (The Project). The Project is funded by the Australian Government through the NDIS Peer Support and Capacity Building Grant. RVA is the national peak body for Australians living with a rare disease.
Introducing MitoCast: your audio learning channel
We know that fatigue, vision changes and cognitive challenges can make it difficult to read or absorb written information. That’s why we’ve launched MitoCast to share Mito Foundation resources in a new, easy-to-understand audio learning format.
This season has 8 episodes, covering topics such as:
- Understanding mito symptoms
- Building a coordinated healthcare team
- Genetics and diagnosis
- Preparing for medical appointments
- Managing fatigue, exercise and mental health.
Listen anytime on our website or on your favourite podcast platform. You can listen in your own time—during a walk, while resting, or on the way to appointments.
That's a wrap on The Bloody Long Walk 2025
- 10 events
- 15,834 walkers
- $2.69 million funds raised
Thank you for all the ways you showed up and spread the word about the mito movement. Next year's dates will be released at the end of January, so follow The Bloody Long Walk on Instagram or Facebook for updates.
Global progress: first medicines approved for mito
The FDA has recently approved 2 new medicines for specific types of mitochondrial disease:
- Forzinity (Elamipretide)
Approved in September for Barth syndrome. - Kygevvi (nucleoside therapy)
Approved in November for Thymidine Kinase 2 deficiency (TK2d).
These approvals are a milestone in the worldwide effort to develop effective treatments — and one day, a cure. We celebrate alongside the scientists, health professionals, and families who contributed to this progress.
What this means for Australians
FDA approval does not mean the medicines are immediately available here. In Australia, new medicines go through:
- Registration by the Therapeutic Goods Administration (TGA)
- Reimbursement assessment through the Pharmaceutical Benefits Scheme (PBS)
If you or someone you care for lives with Barth syndrome or TK2d, we encourage you to speak with your mito specialist about these new therapies and any clinical studies that may be available. Mito Foundation is already working closely with both medicine companies to encourage applications for approval in Australia.
mitoHOPE's first licence
Australia has reached a critical milestone on the path to offering mitochondrial donation. A new licence will allow the mitoHOPE Program to begin training embryologists in this groundbreaking technique using donated human eggs.
This licence is the first of several needed before clinical studies can begin. It’s an important step, but just one phase of a larger, carefully regulated process. Mito Foundation will keep the community informed and supported as we move into this next phase.
Learn more about this licence and what’s still to come in this news update. You can also explore reproductive options or access tailored support by calling the Mito Foundation Helpline 1300 977 180 (option 1).
Looking after yourself during the holidays
The end of the year can be joyful - and draining. Many people in our community have shared the strategies that help them balance energy, connection and rest during the warmer months.
Here are a few gentle reminders:
Plan for rest
Build quiet time into your holiday plans. It's okay to say no.
Keep cool
Heat can increase fatigue. Use fans, cooling towels, or rest indoors when needed.
Focus on mental wellbeing
Explore our mental health and mito resource for tips, tools, and specialist support options shared by people with lived experience.
Stay connected
If this time of year feels tough, reach out to someone you trust.
