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 | ||||