You may view recordings of the 2023 Medical Symposium presentations by clicking on the links below.
Topic | Speaker | Presentation Link | |
Krabbe Disease & RUSP History | Dr. Dietrich Matern | Presentation link coming soon | |
Krabbe Newborn Screening Council | Dr. Joanne Kurtzberg | Presentation link coming soon | |
State NBS Updates Panel | Presentation link coming soon | ||
New York | Dr. Joseph Orsini | ||
Illinois | Dr. Joshua Baker | ||
Tennessee | Gwendolyn McKee | ||
Ohio | Dr. Margie Ream | ||
Pennsylvania | Pat Edge | ||
Kentucky | Dr. Dietrich Matern | ||
Indiana | Jennifer Weaver | ||
Georgia | Dr. Stephanie Keller | ||
New Jersey | Caitlin Russo | ||
South Carolina | Beth Bair | ||
Update on HSCT for Krabbe Disease | Dr. Joanne Kurtzberg | Presentation link coming soon | |
Update on Gene Therapy for Krabbe Disease | Dr. Maria Escolar | Presentation link coming soon | |
NBS Positive for Krabbe Disease - What now? | Dr. Robert Stone | Presentation link coming soon | |
Panel Discussion of NBS for Krabbe Disease: Sensitivity vs. Specificity, Cure vs. Chance of Life | Dr. Dietrich Matern | Presentation link coming soon | |
Update on Newborn Screening for ALD | Dr. Joshua Bonkowsky | https://youtu.be/B39sEURyqLo | |
Update on NBS for MLD and CTX | Dr. Michael Gelb | https://youtu.be/VIAE2vyd45M | |
Leukodystrophy Care Network | |||
LCN Phase II - Current gaps and unmet needs in the Leukodystrophy Community | Anna Grantham | ||
Transitioning into Adulthood: Pitfalls and Opportunities | Catie Becker | https://youtu.be/gVsAP0Bja1c | |
Musculoskeletal CPG | Dr. Sally Evans | https://youtu.be/1XU-z9l32xo | |
Pulmonary CPG | Dr. Geovanny Perez | https://youtu.be/2rn_YevbQU0 | |
The genetics of cerebrotendinous xanthomatosis and implications for NBS | Dr. Austin Larson | https://youtu.be/EsvAFBgBJJA | |
Updates on Treatments and Clinical Trials for Leukodystrophies | |||
DUOC: A Cord Blood Therapy to Myelinate the Brain | Dr. Joanne Kurtzberg | https://youtu.be/osYPY4Ykadc | |
A Clinical Trial of Leriglitazone in Adult Cerebral Adrenoleukodystrophy | Dr. Ali Fatemi | https://youtu.be/ngGPpS7B5tQ | |
Orchard Therapeutics | Angela Tom | In Person Only | |
Vigil Therapeutics - CSF1R | Dr. Andreas Meier | https://youtu.be/G3hvx7J7dnA | |
Clinical Trials in the Ultra Rare Leukodystrophies | Dr. Amanda Nagy | https://youtu.be/LxgKtnSDOaQ |