Adjunct MLD Scientific Meeting

“Integration of the patient perspective into therapy development for MLD”

Friday, November 18th, 2022, from 11 am – 1 pm ET

Please join us as key MLD clinical and research experts take a deep dive into issues identified by the families during October’s EL-PFDD meeting.

On Friday, October 21st, the MLD community hosted an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting. EL-PFDD meetings give the FDA, researchers, biopharma companies, health care providers, and federal regulatory partners an  opportunity to hear directly from patient families. The MLD EL-PFDD featured patient family voices from around the United States, from all subtypes of MLD, and who have experienced various treatment options.

The MLD Scientific Workshop is free and open to the public.  Simply click the button below or visit on November 18th at 11am EST  to view the meeting.  It will be recorded and available for viewing after it premieres.


  • Laura Adang, MD, PhD, Children’s Hospital of Philadelphia

  • Adeline Vanderver, MD, Children’s Hospital of Philadelphia

  • Barbara Burton, MD, Lurie Children’s Hospital

  • Marc Patterson, MD, Mayo Clinic

  • Paul Orchard, MD, University of Minnesota Medical Center

  • Samuel Gröeschel, MD, PhD, University Children’s Hospital Tüebingen


11:00 am – Opening Remarks – Maria Kefalas & James Valentine

11:05 am – Recap of EL-PFDD: What’s Important to MLD Patients/Caregivers – Larry Bauer

11:20 am – Framework for Integrating the Patient Voice into Drug Development – James Valentine

11:30 am – Landscape Analysis of Tools to Evaluate What’s Important to MLD Patients – Dr. Laura Adang

11:45 am – Discussion: Setting a Patient-Focused Research Agenda for Drug Development Tools

12:30 pm – Opportunities to Conduct Clinical Trials in Late-Onset MLD

12:40 pm –Discussion: Perspectives on Facilitating Clinical Trials in Late-Onset MLD

12:55 pm – Closing Remarks – James Valentine

Watch the MLD Research/Scientific Clinical response meeting below:

Use the Chapter Selector in the lower right corner
of the above video window to jump to specific points in the video.