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EL-PFDD Meeting on GSD1a

On October 9, 2025, The Children's Fund for GSD Research hosted an Externally Led Patient Focused Drug Development Meeting (EL-PFDD). This meeting gave patients and families an opportunity to share their voices and stories with the purpose of accelerating research and improving care.
 

EL-PFDD meetings give the Food and Drug Administration (FDA) and other key stakeholders - including researchers, biopharma companies, healthcare providers, federal regulatory partners, and the general public - an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients' daily lives, patients' experiences with currently available treatments, and patient's priorities for therapeutic outcomes.

Agenda & Speakers

  • 10:00AM - 10:05AM

  • Opening Remarks / Welcome

    Meredith Gussin & Lisa Hodes

    Board of Directors

    The Children's Fund for GSD Research

  • 10:05AM - 10:15AM

  • FDA Opening Remarks

    Jemila Venner-Walcott, MD, Office of Therapeutic Products,
    Center for Biologics Evaluation and Research, U.S. Food and Drug Administration

  • 10:15AM - 10:30AM

  • Clinical Overview of GSD1a

    David Rodriguez-Buritica, MD, Geneticist/Endocrinologist and
    Co-Director of Glycogen Storage Disease Clinic at University of Texas at Health Science Center

     

  • 10:30AM - 10:35AM

  • Introduction & Meeting Overview

    James Valentine, JD, MHS, Meeting Moderator

  • 10:35AM - 10:45AM

  • Demographic Polling

    James Valentine, MD, MHS, Meeting Moderator

  • 10:45AM - 11:10AM

  • Patient / Caregiver Panel - Living with GSD1a

    A panel of 5 Patients and Caregivers will provide comments to start the discussion on
    the symptoms and daily impact of living with GSD1a.

  • 11:10AM - 12:30PM

  • Large Group Faciliated Discussion

    Facilitated by Meredith Gussin and James Valentine

    Patients and Caregivers in the audience are invited to add to the dialogue.

  • 12:30 PM - 1:00 PM
  • Break
  • 1:00 PM - 1:10 PM

  • Treatment of GSD1a: An Overview

    Rebecca Riba-Wolman, MD, Pediatric Endocrinologist,
    Director of Glycogen Storage Disease Program & Disorders of Hypoglycemia at Connecticut Children’s

  • 1:10 PM - 1:35 PM

  • Patient / Caregiver Panel - Current and Future Treatments for GSD1a

    A panel of 5 Patients and Caregivers will provide comments to start the discussion on
    GSD1a treatments and treatment experiences.

  • 1:35 PM - 2:45 PM

  • Large-Group Facilitated Discussion

    Facilitated by Meredith Gussin and James Valentine

    Patients and Caregivers in the audience are invited to add to the dialogue.

     

  • 2:45 PM - 2:55 PM

  • Meeting Summary

    Sarah Wicks, JD, MPH

    Hyman, Phelps, & McNamara, P.C. 

  • 2:55 PM - 3:00 PM

  • Closing Remarks 

    Meredith Gussin

    Board of Directors

    The Children's Fund for GSD Research

     

Community Webinar PFDD Q&A

In this Community Webinar, James Valentine and Sarah Wicks of Hyman Phelps McNamara helped families unterstand the drug development process, the role of the FDA and what to expect during the Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting where we informed the U.S. Food and Drug Administration (FDA) and other stakeholders about the patient and caregiver perspectives on living with GSD1a.

 

 

 

ANCHOR. Name: Livestream
ANCHOR. Name: Meeting Summary

Meeting Summary / Voice of the Patient Report

Following the EL-PFDD Meeting on October 9, 2025, we will compile a report in accordance with the FDA's guidance. It will be available to download for free, here. 

 

Questions or comments, please email us at pfdd@curegsd.org.

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