Mitochondrial Disease Can’t Wait for Washington to Catch Up

Mitochondrial disease occurs when the mitochondria in a patient’s cells fail to convert food and oxygen into the energy the body needs to survive – a failure that can affect any organ system and rob a patient of the ability to see, hear, walk, talk, eat, and breathe. It’s estimated to affect 1 in 5,000 people. Many forms are devastatingly progressive, meaning some affected children do not survive beyond their teenage years. Adult-onset disease can bring rapid physical decline. Less than one percent of the 300-plus known genetic variants of mitochondrial disease currently have an FDA-approved treatment.

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1 in 5,000

people are affected by mitochondrial disease

80,000+

estimated people affected in the United States

<1%

of 300+ known genetic variants have an FDA-approved treatment

Our advocates showing up on Capitol Hill – because policy changes when patients and families are in the room.

Our Advocacy Priorities

Where the Coalition Is Focused – and Why It Can’t Wait

Every priority below is a lever we can move together. The faster we move it, the sooner patients feel the difference.

Research Funding

Creating a Robust Environment for Federally-Funded Mitochondrial Disease Research

We support a robust environment for mitochondrial disease research, championing programs like the Peer-Reviewed Medical Research Program and pressing for stronger NIH language and funding.

Legislative

Advancing Legislation That Serves Mitochondrial Disease Families, Clinicians and Researchers

We champion legislation that directly serves mitochondrial disease patients and the clinical and scientific communities who support them.

Regulatory

Supporting a Responsive Regulatory Path for Mitochondrial Disease Treatments

We support a healthy regulatory environment for mitochondrial disease therapies so that promising treatments reach the patients who are waiting for them.

United We’re Louder

A Coalition Built by the Mitochondrial Disease Community

These organizations are standing together to speak with one voice on Capitol Hill – amplifying decades of patient, family, clinician, and researcher advocacy.