Please note, this program is subject to change and will be finalized a week prior to the meeting.

Monday, July 17th
StartFinishTopic/EventSpeakerLocation
All DayCheck inTamarack Lobby
Dinner on your own
Tuesday, July 18th
BreakfastSunrise, 1st Floor HV Lodge
8:00am5:00pmMedical Symposium, Day OneFairway, 2nd Floor HV Lodge
WelcomeFairway, 2nd Floor HV Lodge
Krabbe Disease & RUSP HistoryDr. Dietrich MaternFairway, 2nd Floor HV Lodge
State UpdatesFairway, 2nd Floor HV Lodge
New YorkDr. Joseph OrsiniFairway, 2nd Floor HV Lodge
MissouriFairway, 2nd Floor HV Lodge
IllinoisFairway, 2nd Floor HV Lodge
TennesseeGwendolyn McKeeFairway, 2nd Floor HV Lodge
OhioFairway, 2nd Floor HV Lodge
PennsylvaniaPat EdgeFairway, 2nd Floor HV Lodge
KentuckyFairway, 2nd Floor HV Lodge
IndianaJennifer WeaverFairway, 2nd Floor HV Lodge
GeorgiaFairway, 2nd Floor HV Lodge
New JerseyCaitlin VailFairway, 2nd Floor HV Lodge
South CarolinaFairway, 2nd Floor HV Lodge
Update on HSCT for Krabbe Disease: Dr. Joanne KurtzbergFairway, 2nd Floor HV Lodge
Update on Gene Therapy for Krabbe DiseaseDr. Maria EscolarFairway, 2nd Floor HV Lodge
LunchSunrise, 1st Floor HV Lodge
NBS Positive for Krabbe Disease - What now?Dr. Robert StoneFairway, 2nd Floor HV Lodge
Panel Discussion of NBS for Krabbe Disease: Sensitivity vs. Specificity, Cure vs. Chance of Life Dr. Dietrich MaternFairway, 2nd Floor HV Lodge
Update on Newborn Screening for ALDFairway, 2nd Floor HV Lodge
Update on NBS for MLDDr. Michael GelbFairway, 2nd Floor HV Lodge
Update on NBS for CTXDr. Michael GelbFairway, 2nd Floor HV Lodge
DiscussionFairway, 2nd Floor HV Lodge
DinnerSunrise, 1st Floor HV Lodge
Wendesday, July 19th
BreakfastSunrise, 1st Floor HV Lodge
8:00am4:00pmMedical Symposium, Day TwoFairway, 2nd Floor HV Lodge
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
CTX CPGDr. 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 Tom
Clinical Trials in the Ultra Rare LeukodystrophiesDr. Amanda Nagy
Travere - CTX
Vigil Therapeutics - CSF1RDr. Andreas Meier