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NDC Symposium

Agenda

7:45 AM - 8:00 AM
Opening Comments/Introduction
Bernard L. Maria, MD, MBA, Morristown, NJ

8:00 AM - 10:20 AM
SESSION I: Clinical Aspects and Diagnosis
Co-Director and Moderator: Sakkubai Naidu, MD;
Johns Hopkins University, Baltimore, MD

8:00 AM - 8:20 AM
Overview of Childhood Leukodystrophies
Sakkubai Naidu, MD; Johns Hopkins University, Baltimore, MD

8:20 AM - 8:50 AM
Update on tRNA related Leukoencephalopathies
Marjo van der Knaap, MD; Vrije Universiteit,
Amsterdam, Netherlands

8:50 AM - 9:20 AM
Update on Hypomyelinating Leukodystrophies
Genevieve Bernard, MD; McGill University, Montreal, Canada

9:20 AM - 9:50 AM
Leukoencephalopathies with Brain Calcifications
Yanick Crow, PhD; University of Manchester,
Manchester, UK

9:50 AM - 10:20 AM
Early Diagnosis in Leukodystrophy
Adeline Vanderver, MD; Children’s Hospital of Philadelphia,
Philadelphia, PA

10:20 AM - 10:30 AM
Question and Answer Session

10:30 AM - 10:45 AM
Coffee Break

10:45 AM - 12:35 PM
SESSION II: Pathogenesis
Co-Director and Moderator: Gustavo Maegawa, MD, PhD; University of Florida. Gainesville, FL

10:45 AM - 11:30 AM
Overview of Pathogenesis and Molecular Aspects and Updates on Krabbe Disease
Gustavo Maegawa, MD, PhD; University of Florida. Gainesville, FL

11:30 AM - 11:35 AM
Alexander Disease
Albee Messing, VMD, PhD; University of Wisconsin, Madison, WI

11:35 AM - 12:05 AM
Krabbe Disease
Lawrence Wrabetz, MD; State University of New York at Buffalo, Buffalo, NY

12:05 PM - 12:35 PM
Zebrafish in Leukodystrophies
Joshua Bonkowsky, MD, PhD; University of Utah,
Salt Lake City, UT

12:35 PM - 12:45 PM
Question and Answer Session

12:45 PM - 1:45 PM
Lunch with World Leukodystrophy Alliance (WLA) and Leukodystrophy Care Network (LCN) Presentations

1:45 PM - 4:05 PM
SESSION III: Therapy
Co-Director and Moderator: Ali Fatemi, MD;
Johns Hopkins University, Baltimore, MD

1:45 PM - 2:15 PM
Overview of Therapeutic Approaches
Ali Fatemi, MD; Johns Hopkins University, Baltimore, MD

2:15 PM - 2:50 PM
Gene Therapy
Florian Eichler, MD; Harvard University, Boston, MA

2:50 PM - 3:25 PM
Multimodal Therapy
Mark Sands, PhD; Washington University, St. Louis, MO

3:25 PM - 4:00 PM
Nanotherapy
Sujantha Kannan, MD; Johns Hopkins University, Baltimore, MD

4:05 PM - 4:20 PM
EXECUTIVE SUMMARY OF THE DAY & In-Room Break
Co-Directors and Moderators:
  • Sakku Naidu, MD
  • Ali Fatemi, MD
  • Gustavo Maegawa, MD, PhD
4:20 PM - 4:55 PM
SESSION IV: FUTURE DIRECTIONS & Question and Answer Session
Moderator: Bernard L. Maria, MD, MBA
Panelists: Jacque Waggoner, Albee Messing, Marjo van der Knaap, Florian Eichler, Keith Vanharen, Ann Moser

4:55 PM - 5:00 PM
Closing Comments
Bernard L. Maria, MD, MBA


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