UMDF, Hope for PDCD Leaders to Brief Congressional Mitochondrial Disease Caucus on Regulatory Challenges for Rare Disease Therapies
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United Mitochondrial Disease FoundationFeb 16, 2026, 10:00 ET
Congressional Mitochondrial Disease Caucus briefing to explore how FDA drug review processes affect patient care and access for ultra-rare, life-threatening pediatric mitochondrial diseases, using pyruvate dehydrogenase complex deficiency (PDCD) as an example.
WASHINGTON, Feb. 16, 2026 /PRNewswire-PRWeb/ -- During Rare Disease Week, the , in coordination with the Congressional Mitochondrial Disease Caucus and families from Hope for PDCD, will host a bipartisan congressional briefing with members of Congress and senior congressional staff for the Congressional Mitochondrial Disease House Caucus on Tuesday, February 24, using as an example to explore how FDA drug review processes affect patient care and access for ultra-rare, life-threatening pediatric mitochondrial diseases.
Titled "From Discovery to Delivery: What the FDA Drug Review Process Means for Rare Disease Patients and Providers," the briefing will focus on how FDA approvals – and denials – highlight both progress and persistent challenges in developing treatments for ultra-rare conditions.
Joining UMDF will be a regulatory expert and three caregivers to children with PDCD, a rare and life-threatening neurodegenerative mitochondrial disease. They'll highlight the urgent, unmet need for an FDA-approved treatment, how delays can cause irreversible harm, and how Congress can help ensure the regulatory process reflects the urgency and flexibility needed for their families.
WHEN:
Tuesday, February 24, 2026
2:00 – 3:00 PM ET
WHERE:
Rayburn House Office Building, Room 2060
5 Independence Ave SW
Washington, DC 20515
WHO YOU WILL HEAR FROM:
- Opening Remarks: Congressional Mitochondrial Disease Caucus Co-Chairs The Honorable Brian Fitzpatrick and The Honorable James McGovern
- Mitochondrial Disease Landscape & Progress: Kristen Clifford, President & CEO, United Mitochondrial Disease Foundation
- Regulatory Perspectives: Regulatory experts from Holland & Knight on FDA approval pathways and expedited review processes for serious and rare diseases
- Clinical Perspectives: Rebecca Ganetzky, MD, Children's Hospital of Philadelphia, on the clinical impact of regulatory timelines for patients and providers
- Advocacy & Family Perspectives: Frances Pimentel, Founder & CEO, , and patient and caregiver advocates on the human consequences of regulatory delays for ultra-rare diseases
Media Note: This event is open to the media but registration is required. Please contact [email protected] to confirm attendance.
About United Mitochondrial Disease Foundation (UMDF)
For 30 years, the United Mitochondrial Disease Foundation has brought together a global network of patients, researchers, clinicians, institutions, and industry partners dedicated to fighting mitochondrial disease. Together with the mito community, UMDF utilizes a four-pronged strategy of supporting and connecting affected families, helping train and educate the medical community, working with policymakers for mito-friendly legislation, and helping find research dollars to unlock the science needed for treatments and eventual cures. To learn more, visit .
Media Contact
Andy Dearth, United Mitochondrial Disease Foundation, 1 412-744-1055, [email protected],
SOURCE United Mitochondrial Disease Foundation
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