UMDF Connect
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...
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...
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.
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...
Advocacy Update – December 2023
UMDF Advocacy Update – Dec. 2023 The House is scheduled to be out of session until January, while the Senate is scheduled to remain in session this week to continue to work toward an agreement on funding for Ukraine, Israel, and US border reform, as well as...
A message from UMDF President & CEO Brian Harman regarding STRIDE Study Results from Reneo Pharmaceuticals
To the mitochondrial disease community, We are saddened by the findings recently released by Reneo Pharmaceuticals regarding the STRIDE study for primary mitochondrial myopathy. We know such news weighs heavily on the entire mitochondrial disease community and we...
PDCD FDA Listening Session Report Now Available
On September 8, 2023, the United Mitochondrial Disease Foundation co-hosted a Patient-led Listening Session with the U.S. Food & Drug Administration for Pyruvate Dehydrogenase Complex Deficiency (PDCD) with fellow patient advocacy groups MitoAction, Hope for PDCD,...
Mitochondrial Medicine Society Issues Health Guidance for this Winter
Published 12/5/23, 5:31pm The Mitochondrial Medicine Society (MMS) issued the following statement regarding vaccinations this winter: The changing seasons bring the risk of respiratory seasonal illnesses in our mitochondrial disease patients. Some patients are at...
Ask the Mito Doc – November 2023; Q&A
Ask the Mito Doc November 2023 Coffee and Conversations - Four Breakout Room Sessions DOWNLOAD the Coffee and Conversations Resources (pdf) >> Adult Patients Breakout #1: Mito 101 - Things I Wish I Knew Years AgoFernando Scaglia, MDParents/Caregivers...