New coalition brings together organizations from across the mitochondrial disease community to accelerate research funding, advance patient-centered legislation, and foster a responsive regulatory environment for new therapies.
Seventeen mitochondrial disease-focused organizations in the United States today announced the launch of the Mitochondrial Disease Advocacy Coalition, a unified body formed to speak with one voice on Capitol Hill on behalf of the estimated 80,000 people living with mitochondrial disease in the U.S.
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. 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. There are no cures.
The announcement comes as the community prepares to mark World Mitochondrial Disease Week Sept.14 – Sept. 20, a time that unites patients, families, researchers, clinicians, and advocates worldwide to raise awareness and mobilize support for mitochondrial disease patients.
The coalition, which is being convened by the United Mitochondrial Disease Foundation (UMDF), includes the following organizations:
ATAD3A Patient Advocacy Alliance
Friedreich’s Ataxia Research Alliance (FARA)
Jeremiah Gracen TK2d Foundation
The Elizabeth Watt PDCD Research Fund
“Our community has never lacked passionate advocates. It was simply that the advocacy was directed in a lot of different directions, spread across hundreds of diseases,” said Kristen Clifford, President & CEO of the United Mitochondrial Disease Foundation. “Individually, each organization has fought hard for this community, but no single organization can carry that weight, or command the kind of attention we need on Capitol Hill, alone. This coalition harnesses our collective passion into a single, united voice.”
The group will focus its efforts on three federal priorities: securing and expanding federal research funding for mitochondrial disease; advancing legislative priorities that directly affect mitochondrial disease families; and fostering a regulatory environment that meets the needs and urgency of the community.
The public is encouraged to visit www.mitoadvocacy.org to learn more about the coalition’s priorities, member organizations, and take action on a number of advocacy items.