How to participate in the EL-PFDD

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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