You may view recordings of the 2023 Medical Symposium presentations by clicking on the links below.

TopicSpeakerPresentation Link
Krabbe Disease & RUSP HistoryDr. Dietrich MaternPresentation link coming soon
Krabbe Newborn Screening CouncilDr. Joanne KurtzbergPresentation link coming soon
State NBS Updates PanelPresentation link coming soon
New YorkDr. Joseph Orsini
IllinoisDr. Joshua Baker
TennesseeGwendolyn McKee
OhioDr. Margie Ream
PennsylvaniaPat Edge
KentuckyDr. Dietrich Matern
IndianaJennifer Weaver
GeorgiaDr. Stephanie Keller
New JerseyCaitlin Russo
South CarolinaBeth Bair
Update on HSCT for Krabbe Disease Dr. Joanne KurtzbergPresentation link coming soon
Update on Gene Therapy for Krabbe DiseaseDr. Maria EscolarPresentation link coming soon
NBS Positive for Krabbe Disease - What now?Dr. Robert StonePresentation link coming soon
Panel Discussion of NBS for Krabbe Disease: Sensitivity vs. Specificity, Cure vs. Chance of Life Dr. Dietrich MaternPresentation link coming soon
Update on Newborn Screening for ALDDr. Joshua Bonkowsky
Update on NBS for MLD and CTXDr. Michael Gelb
Leukodystrophy Care Network
LCN Phase II - Current gaps and unmet needs in the Leukodystrophy CommunityAnna Grantham
Transitioning into Adulthood: Pitfalls and OpportunitiesCatie Becker
Musculoskeletal CPGDr. Sally Evans
Pulmonary CPGDr. Geovanny Perez
The genetics of cerebrotendinous xanthomatosis and implications for NBSDr. Austin Larson
Updates on Treatments and Clinical Trials for Leukodystrophies
DUOC: A Cord Blood Therapy to Myelinate the BrainDr. Joanne Kurtzberg
A Clinical Trial of Leriglitazone in Adult Cerebral AdrenoleukodystrophyDr. Ali Fatemi
Orchard TherapeuticsAngela TomIn Person Only
Vigil Therapeutics - CSF1RDr. Andreas Meier
Clinical Trials in the Ultra Rare LeukodystrophiesDr. Amanda Nagy