A Special World Mitochondrial Disease Week Message from UMDF President & CEO Kristen Clifford

Today marks the official kickoff of World Mitochondrial Disease Week, a time designed to help the public better understand mitochondrial disease – with the hope that increased awareness, attention, and funding will accelerate progress toward effective treatments and cures for our entire community.

This year, our focus is on hope. I know, given recent news regarding denied – or at the very least, delayed – therapies for Barth syndrome and PDCD, hope can feel distant. The pathway to any approved therapy, unfortunately, is never going to be a straight line. There will always be obstacles and setbacks. But with each challenge, this community has shown strength, resilience, and the power of unity.

Within 24 hours of the FDA’s denial of DCA to treat PDCD, five mito groups – the United Mitochondrial Disease Foundation, MitoAction, Cure Mito Foundation, Hope for PDCD, and the Elizabeth Watt PDCD Research Foundation – collaborated on a joint statement and action items for Congress. That level of unity gives me hope.

Within 48 hours of the announcement, more than 2,000 of you had already written to Congress to encourage the FDA to apply flexibility in rare disease approvals. That passion gives me hope.

And your work is turning into results. Earlier this month, the FDA Commissioner agreed to meet directly with Barth syndrome families. Shortly after, the agency announced it would complete its review of elamipretide in a matter of weeks – versus the nearly seven months it had originally proposed.

Despite setbacks, we could still have the first FDA-approved mitochondrial disease therapy as early as the end of this month – with another drug application for TK2d still under review. We are closer to progress than ever before!

I continue to believe we are on the cusp of approved mitochondrial disease therapies. And when that happens, it will be because of you.

Please follow along with us on social media, where we’ll be sharing patient stories, mito facts, World Mitochondrial Disease Week events, and more. We also have a variety of downloadable social media badges and covers you can use to promote awareness.

So this week, let’s raise our voices so the world will know and understand this disease.

Together, we will turn hope into hope delivered.

Sincerely,