Pelizaeus-Merzbacher Disease Patient-led Listening Session
On August 22, 2023, six PMD families from across the country, and Dr. Adeline Vanderver, MD, Children’s Hospital of Philadelphia (CHOP), met with the US Food and Drug Administration (FDA) for a closed session listening session.
Together the group was able to share knowledge with FDA staff on the complex issues of PMD, the various physical manifestations and the body systems that are affected.
The listening session demonstrated the importance of raising awareness among FDA staff about the serious impact of PMD disease manifestations on patients and the current lack of FDA approved treatments.
The families who participated in the Listening Session included those of:
Brian and Dylan Gasperetti, Franklin Square, NY
Brant Dunham, Watson, LA
Jack Leonard, Robbinsville, NJ
Jaden Manley, Mexico, NY
James Erena, Milton, VT
Leonardo Xavier, San Diego, CA
Participants from the FDA were very open to the conversation and confirmed the agency’s commitment to working toward treatments for rare diseases. Discourse followed on the most impactful treatments to address PMD symptoms and risk tolerances of families.
The FDA participants expressed gratitude to PMD families for their bravery and willingness to share their stories.
The slide deck presentation and full session summary are now available for download at https://www.pmdfoundation.org/fda-patient-listening-session.
DISCLAIMER
Discussions in FDA Patient Listening Sessions are informal. All opinions, recommendations, and proposals are unofficial and nonbinding on FDA and all other participants. This report reflects The PMD Foundation’s account of the perspectives of patients and caregivers who participated in the Patient Listening Session with the FDA. To the extent possible, the terms used in this summary to describe specific manifestations of Pelizaeus-Merzbacher Disease, health effects and impacts, and treatment experiences, reflect those of the participants. This report is not meant to be representative of the views and experiences of the entire Pelizaeus-Merzbacher Disease patient population or any specific group of individuals or entities. There may be experiences that are not mentioned in this report.