FDA Patient 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 FDA for a closed session listening session to:

• Share knowledge with FDA staff on the complex issues of PMD, the various physical manifestations and the body systems that are affected. 

• Raise awareness among FDA staff on the:

  • serious impact of PMD disease manifestations on patients,

  • effects on quality of life,

  • current lack of FDA approved treatments,

  • tremendous unmet medical need, and

  • preferences for treatments and outcomes.

This listening session was an important step in our clinical trial readiness. It included a Welcome from PMD Foundation Board Chair, Dave Manley, goals for treatments, a clinician’s overview from Dr. Adeline Vanderver, six patient/caregiver perspectives from PMD families, Q&A session and closing remarks from FDA Patient Affairs. 

About Dr. Adeline Vanderver, MD, Children’s Hospital of Philadelphia (CHOP);
Attending physician in the Division of Neurology, Program Director of the Leukodystrophy Center, Professor of Neurology, Perelman School of Medicine, University of Pennsylvania, Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics

Read more about the patient/caregiver perspectives shared during the FDA Patient Listening Session: