UMDF Elevates TK2d Patient Voices at FDA

 

Pittsburgh, PA (January 31, 2021) – The United Mitochondrial Disease Foundation (UMDF) – in collaboration with MitoAction and TK2 Cures – led an FDA Listening Session to help regulators understand the vital importance of new research and clinical trials.

“Listening sessions like this are an integral part of helping the FDA understand the burden of TK2d,” said United Mitochondrial Disease Foundation President and Chief Executive Officer Brian Harman. “It’s imperative that we continue to lift up the voices of those who know this disease best – patients, their loved ones, researchers, and medical experts. We see this as an important first step in charting a course toward treatments and an eventual cure for TK2d.”

Seven representatives from the TK2d patient community participated in the FDA videoconference, along with clinician Dr. Michio Hirano, MD, of Columbia University.

Many of those present urged the FDA to act with expediency when it comes to potential TK2d treatments.

“I know the process of getting approval for medication. I have studied every aspect of his disease,” said Aneesa, mother of three-year-old Jeremiah, a TK2d patient. “I know the FDA and researchers need time. Sadly, for those with this disease, their fate doesn’t have time.”

UMDF would like to thank the affected individuals and families who bravely shared their stories and Dr. Hirano for his expertise, as well as members of the FDA who listened intently and brought thoughtful questions to better understand the disease.