How One FDA Meeting Gave the MELAS Community a Voice

For families living with MELAS, the emotional toll of mitochondrial disease extends far beyond the diagnosis itself. In this episode of Energy in Action, Marcy Young is joined by PFDD panel participants Gordon, Jackie, and Cheryl to reflect on their experience speaking directly to the FDA about the realities of living with MELAS. Together, they share deeply personal stories about caregiving, advocacy, grief, progression, and the urgent need for better treatments and support for mitochondrial disease families.

The conversation explores what it was like to prepare for such a vulnerable and high-stakes meeting, how the panelists unexpectedly formed lasting bonds through the process, and why sharing the hardest parts of this disease matters. From navigating stroke-like episodes and delayed diagnoses to the emotional impact on siblings and caregivers, this episode offers an honest look at the ripple effects of MELAS — while also highlighting the hope that comes from advocacy, connection, and being heard.

In this episode, you’ll hear:

• What a Patient-Focused Drug Development (PFDD) meeting is and why it matters for rare disease communities

• Gordon’s story of his late wife’s sudden MELAS diagnosis and how MitoAction became a lifeline for his family

• Jackie’s perspective as a sibling advocate supporting her brother TJ through disease progression

• Cheryl’s experience caring for both her husband and son while navigating a devastating diagnosis

• The emotional preparation involved in speaking directly to the FDA about life with MELAS

• Why caregivers, siblings, and family members carry their own unique form of grief

• How advocacy and storytelling can create urgency for treatments, research, and change

• The lasting impact of connection within the mitochondrial disease community

Resources & Ways to Connect

• Visit MitoAction’s Website: https://www.mitoaction.org

Learn More About MitoAction

• Visit MitoAction’s Website: https://www.mitoaction.org

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