How to participate in the EL-PFDD
The EL-PFDD meeting was successfully completed. Please visit this page to view The Voice of the Patient report to see what was shared.
Agenda
Friday Oct. 21st, 2022
10am – 3 pm EDT
10:00 am – 10:05 am
Welcome – Dean Suhr, MLD Foundation
10:05 am – 10:15 am
FDA Opening Remarks – Dr. Sairah Thommi, Division of Clinical Evaluation & Pharmacology/Toxicology OTAT, CBER – FDA
10:15 am – 10:30 am
MLD Clinical Overview – Laura Adang, MD, PhD, Children’s Hospital of Philadelphia
10:30 am – 10:35 am
Introduction and Meeting Overview – James Valentine, JD, MHS, Meeting Moderator
10:35 am – 10:45 am
Demographic Polling
Session 1 – Living with MLD Symptoms and Daily Impact
10:45 am – 11:10 am
Patient/Caregiver Panel 1
11:10 am – 12:30 pm
Audience polling & moderated discussion – including telephone call-ins and written comments
12:30pm – 1:00 pm
Lunch
Session 2 – Current and Future Treatments for MLD
1:00 pm – 1:10pm
Treatment Overview – Adeline Vanderver, MD – Children’s Hospital of Philadelphia
1:10 pm – 1:35 pm
Patient/Caregiver Panel 2
1:35 pm – 2:45 pm
Audience polling & moderated discussion – including telephone call-ins and written comments
2:45 pm – 2:55 pm
Summary Remarks – Larry Bauer, RN, MA
2:55 pm – 3:00 pm
Closing Remarks – Maria Kefalas, PhD, The Calliope Joy Foundation
How and Why You Should Participate in the MLD EL-PFDD
- 0:00 – Welcome and quick overview
- 6:50 – Introductions: James Valentine, JD, MHS & Larry Bauer, MA, RN
- 9:20 – Background on FDA & Drug Development
- 30:41 – Introduction to PFDD / Patient Voice
- 34:52 – Overview of the EL-PFDD meeting
- 36:52 – Guide to participating in the meeting
- 45:00 – Q&A and Discussion