Accessibility Statement Skip Navigation
  • Why PRWeb
  • How It Works
  • Who Uses It
  • Pricing
  • Login
  • GDPR
  • Create a Free Account
Return to PRWeb homepage
  • News
  • Resources
  • Contact
When typing in this field, a list of search results will appear and be automatically updated as you type.

Searching for your content...

No results found. Please change your search terms and try again.
  • News in Focus
      • Browse News Releases

      • All News Releases
      • Multimedia Gallery

      • All Multimedia
      • All Photos
      • All Videos
  • Business & Money
      • Auto & Transportation

      • Aerospace, Defense
      • Air Freight
      • Airlines & Aviation
      • Automotive
      • Maritime & Shipbuilding
      • Railroads and Intermodal Transportation
      • Supply Chain/Logistics
      • Transportation, Trucking & Railroad
      • Travel
      • Trucking and Road Transportation
      • View All Auto & Transportation

      • Business Technology

      • Blockchain
      • Broadcast Tech
      • Computer & Electronics
      • Computer Hardware
      • Computer Software
      • Data Analytics
      • Electronic Commerce
      • Electronic Components
      • Electronic Design Automation
      • Financial Technology
      • High Tech Security
      • Internet Technology
      • Nanotechnology
      • Networks
      • Peripherals
      • Semiconductors
      • View All Business Technology

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film and Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Financial Services & Investing

      • Accounting News & Issues
      • Acquisitions, Mergers and Takeovers
      • Banking & Financial Services
      • Bankruptcy
      • Bond & Stock Ratings
      • Conference Call Announcements
      • Contracts
      • Cryptocurrency
      • Dividends
      • Earnings
      • Earnings Forecasts & Projections
      • Financing Agreements
      • Insurance
      • Investments Opinions
      • Joint Ventures
      • Mutual Funds
      • Private Placement
      • Real Estate
      • Restructuring & Recapitalization
      • Sales Reports
      • Shareholder Activism
      • Shareholder Meetings
      • Stock Offering
      • Stock Split
      • Venture Capital
      • View All Financial Services & Investing

      • General Business

      • Awards
      • Commercial Real Estate
      • Corporate Expansion
      • Earnings
      • Environmental, Social and Governance (ESG)
      • Human Resource & Workforce Management
      • Licensing
      • New Products & Services
      • Obituaries
      • Outsourcing Businesses
      • Overseas Real Estate (non-US)
      • Personnel Announcements
      • Real Estate Transactions
      • Residential Real Estate
      • Small Business Services
      • Socially Responsible Investing
      • Surveys, Polls and Research
      • Trade Show News
      • View All General Business

  • Science & Tech
      • Consumer Technology

      • Artificial Intelligence
      • Blockchain
      • Cloud Computing/Internet of Things
      • Computer Electronics
      • Computer Hardware
      • Computer Software
      • Consumer Electronics
      • Cryptocurrency
      • Data Analytics
      • Electronic Commerce
      • Electronic Gaming
      • Financial Technology
      • Mobile Entertainment
      • Multimedia & Internet
      • Peripherals
      • Social Media
      • STEM (Science, Tech, Engineering, Math)
      • Supply Chain/Logistics
      • Wireless Communications
      • View All Consumer Technology

      • Energy & Natural Resources

      • Alternative Energies
      • Chemical
      • Electrical Utilities
      • Gas
      • General Manufacturing
      • Mining
      • Mining & Metals
      • Oil & Energy
      • Oil and Gas Discoveries
      • Utilities
      • Water Utilities
      • View All Energy & Natural Resources

      • Environ­ment

      • Conservation & Recycling
      • Environmental Issues
      • Environmental Policy
      • Environmental Products & Services
      • Green Technology
      • Natural Disasters
      • View All Environ­ment

      • Heavy Industry & Manufacturing

      • Aerospace & Defense
      • Agriculture
      • Chemical
      • Construction & Building
      • General Manufacturing
      • HVAC (Heating, Ventilation and Air-Conditioning)
      • Machinery
      • Machine Tools, Metalworking and Metallurgy
      • Mining
      • Mining & Metals
      • Paper, Forest Products & Containers
      • Precious Metals
      • Textiles
      • Tobacco
      • View All Heavy Industry & Manufacturing

      • Telecomm­unications

      • Carriers and Services
      • Mobile Entertainment
      • Networks
      • Peripherals
      • Telecommunications Equipment
      • Telecommunications Industry
      • VoIP (Voice over Internet Protocol)
      • Wireless Communications
      • View All Telecomm­unications

  • Lifestyle & Health
      • Consumer Products & Retail

      • Animals & Pets
      • Beers, Wines and Spirits
      • Beverages
      • Bridal Services
      • Cannabis
      • Cosmetics and Personal Care
      • Fashion
      • Food & Beverages
      • Furniture and Furnishings
      • Home Improvement
      • Household, Consumer & Cosmetics
      • Household Products
      • Jewelry
      • Non-Alcoholic Beverages
      • Office Products
      • Organic Food
      • Product Recalls
      • Restaurants
      • Retail
      • Supermarkets
      • Toys
      • View All Consumer Products & Retail

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film and Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Health

      • Biometrics
      • Biotechnology
      • Clinical Trials & Medical Discoveries
      • Dentistry
      • FDA Approval
      • Fitness/Wellness
      • Health Care & Hospitals
      • Health Insurance
      • Infection Control
      • International Medical Approval
      • Medical Equipment
      • Medical Pharmaceuticals
      • Mental Health
      • Pharmaceuticals
      • Supplementary Medicine
      • View All Health

      • Sports

      • General Sports
      • Outdoors, Camping & Hiking
      • Sporting Events
      • Sports Equipment & Accessories
      • View All Sports

      • Travel

      • Amusement Parks and Tourist Attractions
      • Gambling & Casinos
      • Hotels and Resorts
      • Leisure & Tourism
      • Outdoors, Camping & Hiking
      • Passenger Aviation
      • Travel Industry
      • View All Travel

  • Policy & Public Interest
      • Policy & Public Interest

      • Advocacy Group Opinion
      • Animal Welfare
      • Congressional & Presidential Campaigns
      • Corporate Social Responsibility
      • Domestic Policy
      • Economic News, Trends, Analysis
      • Education
      • Environmental
      • European Government
      • FDA Approval
      • Federal and State Legislation
      • Federal Executive Branch & Agency
      • Foreign Policy & International Affairs
      • Homeland Security
      • Labor & Union
      • Legal Issues
      • Natural Disasters
      • Not For Profit
      • Patent Law
      • Public Safety
      • Trade Policy
      • U.S. State Policy
      • View All Policy & Public Interest

  • People & Culture
      • People & Culture

      • Aboriginal, First Nations & Native American
      • African American
      • Asian American
      • Children
      • Diversity, Equity & Inclusion
      • Hispanic
      • Lesbian, Gay & Bisexual
      • Men's Interest
      • People with Disabilities
      • Religion
      • Senior Citizens
      • Veterans
      • Women
      • View All People & Culture

  • Hamburger menu
  • Cision PRWeb provides efficient communication tools to continuously engage with target audiences across multiple online channels
  • Create a Free Account
    • ALL CONTACT INFO
    • Contact Us


      11AM ET Sunday – 8PM ET Friday

  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR
  • News in Focus
    • Browse All News
    • Multimedia Gallery
  • Business & Money
    • Auto & Transportation
    • Business Technology
    • Entertain­ment & Media
    • Financial Services & Investing
    • General Business
  • Science & Tech
    • Consumer Technology
    • Energy & Natural Resources
    • Environ­ment
    • Heavy Industry & Manufacturing
    • Telecomm­unications
  • Lifestyle & Health
    • Consumer Products & Retail
    • Entertain­ment & Media
    • Health
    • Sports
    • Travel
  • Policy & Public Interest
  • People & Culture
    • People & Culture
  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR
  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR
  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR

UMDF, MitoAction Help Lift Up Patient Voices on FDA Listening Session for Pyruvate Dehydrogenase Complex Deficiency (PDCD)


News provided by

United Mitochondrial Disease Foundation

Sep 15, 2023, 10:30 ET

Share this article

Share toX

Share this article

Share toX

PDCD FDA Listening Session
PDCD FDA Listening Session

The United Mitochondrial Disease Foundation and MitoAction share their reactions to a September 8, 2023 listening session with the U.S. Food and Drug Administration regarding Pyruvate Dehydrogenase Complex Deficiency (PDCD).

PITTSBURGH, Sept. 15, 2023 /PRNewswire-PRWeb/ -- Today, patient advocacy groups, the United Mitochondrial Disease Foundation (UMDF) and MitoAction shared reactions from a listening session with the U.S. Food and Drug Administration (FDA) regarding Pyruvate Dehydrogenase Complex Deficiency (PDCD).

The September 8, 2023 closed virtual session, "The Voice of Pyruvate Dehydrogenase Complex Deficiency Patient Community," was requested by members of the PDCD community to help the FDA understand the disease.

"Families are doing everything they can with the resources at their disposal, but this is a devastatingly progressive disease. As we work together to make an approved PDCD treatment a reality, it's vital the process reflects that urgency." -- UMDF President and CEO Brian Harman.

Post this

"The FDA heard courageous testimony about the toll this disease takes on patients, about the need for improved diagnostic and screening processes, and about the lengths caregivers are going to support the health of their loved ones. Above all, they heard about the urgent need for an approved treatment. Families are doing everything they can with the resources at their disposal, but this is a devastatingly progressive disease. As we work together to make an approved PDCD treatment a reality, it's vital the process reflects that urgency," said UMDF President and Chief Executive Officer Brian Harman.

"We thank the FDA for the opportunity to help their staff understand the PDCD community's experiences and perspectives. It's crucial that we continue to lift up the voices of expertise – patients, families, clinicians, and researchers – in forums like this," he added.

Said MitoAction Chief Executive Officer Kira Mann: "As the patient-centered patient advocacy organization, we stand committed to fostering a space where the voices of those affected by mitochondrial diseases like PCDC can resonate. Just as we are dedicated to amplifying these voices, we also acknowledge the paramount significance of empowering patients and caregivers with knowledge about the FDA regulatory process. As advocates, we aim to empower individuals with the tools they need to actively engage in this critical process and ensure their voices are heard, because when patients are informed and engaged, they become a driving force for progress."

UMDF and MitoAction thank the PDCD community members who bravely shared their stories and the many patient advocacy groups that helped make the listening session a success including Hope for PDCD and The Elizabeth Watt PDCD Research Fund.

Six caregivers to PDCD patients (one of whom is also an affected adult) participated in the FDA videoconference, along with clinician Rebecca Ganetzky, MD, of Children's Hospital of Philadelphia.

Amongst the testimony heard was Emma, whose daughter Elizabeth is a 20-year-old patient with PDCD.

"As a family, we have lived in a crisis mode for the past 20 years. Constantly worried about death. We have already watched her regress medically and physically, and we fear that every illness will be the one that ends her life. Even minor illnesses can be catastrophic, so we live all day every day in fear. The sad fact is there is not a definitive treatment for PDCD," she said. "Collectively, we come here today to ask for the hope that all people diagnosed with PDCD will be able to live a long and fulfilling life."

This was the second FDA Listening Session UMDF and MitoAction have cohosted for a mitochondrial disease in the last 20 months. In January 2022, the FDA held a similar session for thymidine kinase 2 deficiency (TK2d), another rare mitochondrial disease.

ABOUT PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY

Pyruvate Dehydrogenase Complex Deficiency (PDCD) is a rare mitochondrial disorder that causes impaired carbohydrate metabolism, resulting in neurological problems and a buildup of a chemical called lactic acid in the blood. PDCD is caused by mutations in the genes that make up or affect the activity of the PDC, which is located in the mitochondria that make almost all the energy we need to survive. Nearly all individuals with PDCD experience developmental delay, poor muscle tone, and brain cell loss, which are often accompanied by a host of other issues. Like all mitochondrial diseases, there is currently no FDA-approved treatment or cure for PDCD.

ABOUT THE UNITED MITOCHONDRIAL DISEASE FOUNDATION

Founded in 1996, the United Mitochondrial Disease Foundation (UMDF) works to promote research and education for the diagnosis, treatment and cure of mitochondrial diseases and to provide support for affected individuals and families. Since its inception, the UMDF has funded more than $15 million in research, making it the leading non-governmental contributor of grants focused solely on mitochondrial disease. Every year, UMDF provides support and education to thousands of patients and medical professionals across the globe. Learn more at umdf.org.

ABOUT MITOACTION

Since 2005, MitoAction has transformed the lives of families affected by mitochondrial disease. Our mission is to improve the quality of life for children, adults, and families living with mitochondrial disease through support, education, outreach, advocacy, clinical research initiatives, and granting wishes for children affected by mitochondrial disease. Committed to making the largest impact possible, MitoAction serves individuals in the U.S. and worldwide through support, education, outreach, advocacy, and clinical research initiatives. The programs and services MitoAction provides continue to be a lifeline for families impacted by mitochondrial disease. Families know with MitoAction, they will never be alone on this difficult journey. Learn more at mitoaction.org.

Media Contact

Andy Dearth, United Mitochondrial Disease Foundation, 1 4129799833, [email protected], umdf.org

SOURCE United Mitochondrial Disease Foundation

Modal title

Contact PRWeb

  • 11AM ET Sunday – 8PM ET Friday
  • Contact Us

About PRWeb

  • About PRWeb
  • Partners
  • Partnership Programs
  • Editorial Guidelines
  • Resources

Why PRWeb

  • Why PRWeb
  • How It Works
  • Who Uses It
  • Pricing

Accounts

  • Create a Free Account
  • Log in
  • Contact Us

Do not sell or share my personal information:

  • Submit via [email protected] 
  • Call Privacy toll-free: 877-297-8921

Contact Cision

Products

About

My Services
  • All News Releases
  • Online Member Center
  • ProfNet
Cision Distribution Helpline
888-776-0942
  • Legal
  • Site Map
  • RSS
  • Cookie Settings
Copyright © 2025 Cision US Inc.