UMDF Connect
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...
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...
Advocacy Update – November 2023: CMS Changes Definition of Medicare Caregiver in New Rule
Published 11/29/23 President Biden Signs Funding Bill, Averting a Government Shutdown On November 16, President Biden signed the stopgap funding bill passed by the House and Senate. The short-term two-tiered spending bill includes temporary extensions of expiring...