To the mitochondrial disease community,
As I wrap up my first full week as United Mitochondrial Disease Foundation (UMDF) President and Chief Executive Officer, I wanted to take a moment to reach out.
In my brief time here, I have already had the privilege of meeting some of you, including patients, families, and volunteers. I am humbled by the stories you have shared. In your words, it’s easy to hear the passion, determination, and resilience this community shares — the very traits that built UMDF and have kept this organization going for almost three decades. This organization is truly a reflection of you.
As UMDF’s leader, my commitment is our team will share that same intensity when serving you – be it finding you the support your family needs, funding research crucial to unlocking treatments, advocating for legislation on Capitol Hill, or educating the medical community and the world about this disease.
In these initial days, I have also had the pleasure of speaking with some of the leading researchers and clinicians in the mitochondrial disease space. From those conversations, it’s clear this community is on the precipice of incredible things when it comes to the therapeutic landscape. There are nearly a dozen potential treatments in the therapeutic pipeline worldwide, a number I’m sure would have sounded unbelievable just a decade ago. Along with those potential advancements, comes the need for us to support the patient journey and examine barriers to high quality care that UMDF can positively impact.
The FDA could soon give their approval for a form of mitochondrial disease – in this case, elamipretide for use by Barth Syndrome patients. While the agency’s decision would be limited to that narrow indication, the approval alone has the power to change the complexion of the entire mitochondrial disease therapeutic landscape, bringing a groundswell of interest from researchers and industry.
And lest you think we’re pinning our hopes on this single decision, know it’s not just this therapy. We expect many more New Drug Applications to be filed with the FDA in the coming months and years. Thank you, to each of you — all the patients, families, volunteers, donors, clinicians, and researchers in the mitochondrial community for your unwavering dedication and invaluable contributions. Your collective efforts have brought us to this moment of hope, where potential treatments are within reach, offering brighter futures for so many.
As a transplanted Austin, Texas resident and music lover, it seems appropriate to quote Mr. Bob Dylan: “For the times they are a-changin’.” My promise to you is we will fight together until that change —access to a treatment, high quality care, and ultimately a cure — has come for every mitochondrial disease patient.
Sincerely,
Kristen Clifford
President & CEO, United Mitochondrial Disease Foundation