Please note, this program is subject to change and will be finalized a week prior to the meeting.
Monday, July 17th | |||||
Start | Finish | Topic/Event | Speaker | Location | |
All Day | Check in | Tamarack Lobby | |||
Dinner on your own | |||||
Tuesday, July 18th | |||||
Breakfast | Sunrise, 1st Floor HV Lodge | ||||
8:00am | 5:00pm | Medical Symposium, Day One | Fairway, 2nd Floor HV Lodge | ||
Welcome | Fairway, 2nd Floor HV Lodge | ||||
Krabbe Disease & RUSP History | Dr. Dietrich Matern | Fairway, 2nd Floor HV Lodge | |||
State Updates | Fairway, 2nd Floor HV Lodge | ||||
New York | Dr. Joseph Orsini | Fairway, 2nd Floor HV Lodge | |||
Missouri | Fairway, 2nd Floor HV Lodge | ||||
Illinois | Fairway, 2nd Floor HV Lodge | ||||
Tennessee | Gwendolyn McKee | Fairway, 2nd Floor HV Lodge | |||
Ohio | Fairway, 2nd Floor HV Lodge | ||||
Pennsylvania | Pat Edge | Fairway, 2nd Floor HV Lodge | |||
Kentucky | Fairway, 2nd Floor HV Lodge | ||||
Indiana | Jennifer Weaver | Fairway, 2nd Floor HV Lodge | |||
Georgia | Fairway, 2nd Floor HV Lodge | ||||
New Jersey | Caitlin Vail | Fairway, 2nd Floor HV Lodge | |||
South Carolina | Fairway, 2nd Floor HV Lodge | ||||
Update on HSCT for Krabbe Disease: | Dr. Joanne Kurtzberg | Fairway, 2nd Floor HV Lodge | |||
Update on Gene Therapy for Krabbe Disease | Dr. Maria Escolar | Fairway, 2nd Floor HV Lodge | |||
Lunch | Sunrise, 1st Floor HV Lodge | ||||
NBS Positive for Krabbe Disease - What now? | Dr. Robert Stone | Fairway, 2nd Floor HV Lodge | |||
Panel Discussion of NBS for Krabbe Disease: Sensitivity vs. Specificity, Cure vs. Chance of Life | Dr. Dietrich Matern | Fairway, 2nd Floor HV Lodge | |||
Update on Newborn Screening for ALD | Fairway, 2nd Floor HV Lodge | ||||
Update on NBS for MLD | Dr. Michael Gelb | Fairway, 2nd Floor HV Lodge | |||
Update on NBS for CTX | Dr. Michael Gelb | Fairway, 2nd Floor HV Lodge | |||
Discussion | Fairway, 2nd Floor HV Lodge | ||||
Dinner | Sunrise, 1st Floor HV Lodge | ||||
Wendesday, July 19th | |||||
Breakfast | Sunrise, 1st Floor HV Lodge | ||||
8:00am | 4:00pm | Medical Symposium, Day Two | Fairway, 2nd Floor HV Lodge | ||
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 | ||||
Musculoskeletal CPG | Dr. Sally Evans | ||||
Pulmonary CPG | Dr. Geovanny Perez | ||||
CTX CPG | Dr. Austin Larson | ||||
Updates on Treatments and Clinical Trials for Leukodystrophies | |||||
DUOC: A Cord Blood Therapy to Myelinate the Brain | Dr. Joanne Kurtzberg | ||||
A Clinical Trial of Leriglitazone in Adult Cerebral Adrenoleukodystrophy | Dr. Ali Fatemi | ||||
Orchard Therapeutics | Angela Tom | ||||
Clinical Trials in the Ultra Rare Leukodystrophies | Dr. Amanda Nagy | ||||
Travere - CTX | |||||
Vigil Therapeutics - CSF1R | Dr. Andreas Meier | ||||