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 Bonkowskyhttps://youtu.be/B39sEURyqLo
Update on NBS for MLD and CTXDr. Michael Gelbhttps://youtu.be/VIAE2vyd45M
Leukodystrophy Care Network
LCN Phase II - Current gaps and unmet needs in the Leukodystrophy CommunityAnna Grantham
Transitioning into Adulthood: Pitfalls and OpportunitiesCatie Beckerhttps://youtu.be/gVsAP0Bja1c
Musculoskeletal CPGDr. Sally Evanshttps://youtu.be/1XU-z9l32xo
Pulmonary CPGDr. Geovanny Perezhttps://youtu.be/2rn_YevbQU0
The genetics of cerebrotendinous xanthomatosis and implications for NBSDr. Austin Larsonhttps://youtu.be/EsvAFBgBJJA
Updates on Treatments and Clinical Trials for Leukodystrophies
DUOC: A Cord Blood Therapy to Myelinate the BrainDr. Joanne Kurtzberghttps://youtu.be/osYPY4Ykadc
A Clinical Trial of Leriglitazone in Adult Cerebral AdrenoleukodystrophyDr. Ali Fatemihttps://youtu.be/ngGPpS7B5tQ
Orchard TherapeuticsAngela TomIn Person Only
Vigil Therapeutics - CSF1RDr. Andreas Meierhttps://youtu.be/G3hvx7J7dnA
Clinical Trials in the Ultra Rare LeukodystrophiesDr. Amanda Nagyhttps://youtu.be/LxgKtnSDOaQ