| All Week | Prayer Room | Everyone Welcome! | Birdie, 2nd Fl HV Lodge | |||
| ALL TIMES ARE IN EASTERN TIME ZONE | ||||||
| Start | Topic/Event | Speaker | Location | |||
| Monday, July 15th | Arrive/Check In | Make sure to grab your name tag and welcome bag in the Tamarack Lobby! | ||||
| Tuesday, July 16th | Medical Symposium, Day One | |||||
| 7:00am | Breakfast | Fairway, 2nd Floor HV Lodge | ||||
| Preparing for Nationwide NBS for Krabbe Disease and other Leukodystrophies | Moderator: Dr. Robert Thompson Stone | Fairway, 2nd Floor HV Lodge | ||||
| 8:00am EST | Welcome | |||||
| 8:20am | - Getting Krabbe Disease on the RUSP and its possible consequences | Dr. Dietrich Matern | ||||
| 8:50am | Panel Discussion: State NBS Programs | Moderated by Dr. Dietrich Matern | ||||
| Panelists: | ||||||
| Dr. Joseph Orsini, NY | ||||||
| Tracy Klug, MO | ||||||
| Darrin Sevier, KY | ||||||
| Sharon Linard, OH | ||||||
| Gwendolyn McKee, TN | ||||||
| Dr. Barbara Burton, IL | ||||||
| Barb Lesko, IN | ||||||
| Dr. Taraka Donti, PA | ||||||
| Angela Wittenauer, GA | ||||||
| Elizabeth Bair, SC | ||||||
| 10:00am | BREAK | |||||
| 10:15am | Krabbe Disease Newborn Screening Council | Dr. Joe Orsini | ||||
| 10:30am | Panel Discussion: Communicating with a family who has screened positive for Krabbe Disease | Dr. Barbara Burton, Dr. Joanne Kurtzberg, Dr. Robert Thompson Stone | ||||
| 11:30am | Krabbe Disease Transplant Protocols and Outcomes | Dr. Joanne Kurtzberg | ||||
| 12:30pm | Lunch | Sunrise, 1st Floor HV Lodge | ||||
| 1:30pm | Panel Discussion: Preparing for an IKD Referral | Christin Webb, Dr. Joanne Kurtzberg, Dr. Paul Orchard, Dr. Hemalatha Rangarajan, Dr. Jennifer Rubin, Dr. Shalini Shenoy | ||||
| 2:15pm | LOKD Database | Dr. Camille Corre | ||||
| 2:30pm | Phenotype-Genotype Correlations in Krabbe Disease | Dr. Michael Gelb | ||||
| 2:45pm | BREAK | |||||
| 3:00pm | ALD NBS Overview and Update | Dr. Ali Fatemi | ||||
| 3:30pm | MLD NBS Overview and Update | Dr. Michael Gelb and Dr. Barbara Burton | ||||
| 4:00pm | CTX NBS Overview and Update | Dr. Andrea DeBarber and Dr. Michael Gelb | ||||
| 4:35pm | GROUP PHOTO | |||||
| Meeting Ends | ||||||
| 6:00pm | Dinner | Sunrise, 1st Floor HV Lodge | ||||
| Wednesday, July 17th | Medical Symposium, Day Two | |||||
| 7:00am | Breakfast | Fairway, 2nd Floor HV Lodge | ||||
| Leukodystrophy Care Network | Moderator: Dr. Robert Thompson Stone | |||||
| 8:00am EST | Opening Remarks | |||||
| Industry updates | ||||||
| 8:10am | Orchard Therapeutics | Jen Pyron | ||||
| 8:40am | Mirum Pharmaceuticals | Dr. Rana Dutta | ||||
| 9:00am | Vigil Neuroscience | Dr. Petra Kaufmann | ||||
| 9:20am | Forge Biologics | Dr. Maria Escolar | ||||
| 9:50am | Clinical translation of a Krabbe Disease gene therapy without HSC transplant | Dr. Steven Gray | ||||
| 10:20am | BREAK | |||||
| 10:40am | Overview of Vanishing White Matter | Dr. Joshua Bonkowsky | ||||
| 11:40am | Transition of Care | Catie Becker | ||||
| 12:00pm | Lunch | Sunrise, 1st Floor HV Lodge | ||||
| 1:00pm | Panel Discussion: Consensus on long term follow-up for Leukodystrophy NBS patients | Dr. Ali Fatemi, Dr. Joanne Kurtzberg, Dr. James Provenzale, Dr. Margie Ream, Dr. Robert Thompson Stone, Amy White | Fairway, 2nd Floor HV Lodge | |||
| 2:00pm | Leukodystrophy Focused Payer Education | Sarah Waite-Ardini | ||||
| 2:30pm | Next steps for LCN Clinical Practice Guidelines | Dr. Margie Ream | ||||
| 4:00pm | Meeting Closes | |||||
| 6:00pm | Family Welcome Dinner | Sunrise, 1st Floor HV Lodge | ||||
| Click here to view Thursday's agenda | ||||||
