UMDF Connect
Research Update from 2023 accelerators Runner-Up Jonathan Dietz, PhD
Research Update from 2023 accelerators Runner-Up Jonathan Dietz, PhD In June 2023, Jonathan Dietz, PhD presented his research project “The Role of Metaxins in Mitochondrial Health and Homeostasis” during the Big Pitch event at UMDF’s annual conference on...
Meet Gavin
“I can’t even tell you how often we were in the hospital in those early years,” said Nikki about her son Gavin, who was diagnosed with mitochondrial disease as a toddler. The disease rapidly progressed, causing chaos in Gavin’s small body. At age five, he received a...
Meet Jeremiah
Like many parents, Aneesa’s introduction to mitochondrial disease was jarring. After months of jumping from specialist to specialist who ran test after test searching for answers around the weakness in her then seven-month-old son, Jeremiah, she got a dreaded answer....
Mitochondrial Medicine 2023: Rethink Medicaid: Government Programs & Medicaid Waivers
DOWNLOAD Stacy and Jenny's Slides (pdf) >> DOWNLOAD the Medicaid Glossary of Terms (pdf) >> Due to technical issues with the live recording in Charlotte, this presentation has been RE-RECORDED for our community.
Advocacy Update – February 2024: Redefining “Rare” For the World, Funding for 2024 & Beyond
Rare Disease Day On February 29, we'll join the nearly 300 million affected patients and their loved ones across the world in marking Rare Disease Day. It's a day where we collectively raise awareness and generate support for those living with rare disease -- but...
UMDF Family Impact Funds Award $200k Grant to CHOP to Explore Potential Single Large-Scale mtDNA Deletion Syndrome Treatments
The United Mitochondrial Disease Foundation (UMDF) is pleased to announce a new grant of $200,000 awarded to Suraiya Haroon, PhD, within Children’s Hospital of Philadelphia’s (CHOP) Mitochondrial Medicine Frontier Program (MMFP) research group through the generous...
Ask the Mito Doc – February 2024; Q&A
Ask the Mito Doc - February 2024 Q&A All answers today are based on personal experience of the participants. As always, please consult your personal physician prior to taking any action. Clinicians: Todd J. Kilbaugh, MD, Children's Hospital of...
Advocacy Update – January 2024: New FDA Guidance Encouraging Sign for Rare Disease Patients
In a landmark move, in December 2023, the U.S. Food and Drug Administration (FDA) released a detailed guidance document titled “Rare Diseases: Considerations for the Development of Drugs and Biological Products.” The FDA guidance document is a significant stride...
Ask the Mito Doc – January 2024; Q&A
Ask the Mito Doc - January 2024 Q&A All answers today are based on personal experience of the participants. As always, please consult your personal physician prior to taking any action.Clinicians: Todd J. Kilbaugh, MD, Children's Hospital of Philadelphia,...
Ask the Mito Doc – December 2023; Q&A
Ask the Mito Doc - December 2023 Q&A Wrapping Up 2023 with the Mito Docs Clinicians: Dr. Amy Goldstein, MD, Children’s Hospital of Philadelphia Dr. Austin Larson, MD, Children’s Hospital, Colorado Dr. Divakar Mithal, MD, Children’s Hospital, Chicago...