All WeekPrayer RoomEveryone Welcome!Birdie, 2nd Fl HV Lodge
Monday, July 15thArrive/Check InMake sure to grab your name tag and welcome bag in the Tamarack Lobby!
Tuesday, July 16thMedical Symposium, Day One
7:00amBreakfastFairway, 2nd Floor HV Lodge
Preparing for Nationwide NBS for Krabbe Disease and other LeukodystrophiesModerator:
Dr. Robert Thompson Stone
Fairway, 2nd Floor HV Lodge
8:00am ESTWelcome
8:20am- Getting Krabbe Disease on the RUSP and its possible consequencesDr. Dietrich Matern
8:50amPanel Discussion: State NBS Programs Moderated by Dr. Dietrich Matern
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
10:15amKrabbe Disease Newborn Screening CouncilDr. Joe Orsini
10:30amPanel Discussion: Communicating with a family who has screened positive for Krabbe DiseaseDr. Barbara Burton, Dr. Joanne Kurtzberg, Dr. Robert Thompson Stone
11:30amKrabbe Disease Transplant Protocols and OutcomesDr. Joanne Kurtzberg
12:30pmLunchSunrise, 1st Floor HV Lodge
1:30pmPanel Discussion: Preparing for an IKD ReferralChristin Webb, Dr. Joanne Kurtzberg, Dr. Paul Orchard, Dr. Hemalatha Rangarajan, Dr. Jennifer Rubin, Dr. Shalini Shenoy
2:15pmLOKD DatabaseDr. Camille Corre
2:30pmPhenotype-Genotype Correlations in Krabbe DiseaseDr. Michael Gelb
3:00pmALD NBS Overview and UpdateDr. Ali Fatemi
3:30pmMLD NBS Overview and UpdateDr. Michael Gelb and Dr. Barbara Burton
4:00pmCTX NBS Overview and UpdateDr. Andrea DeBarber and Dr. Michael Gelb
Meeting Ends
6:00pmDinnerSunrise, 1st Floor HV Lodge
Wednesday, July 17thMedical Symposium, Day Two
7:00amBreakfastFairway, 2nd Floor HV Lodge
Leukodystrophy Care NetworkModerator:
Dr. Robert Thompson Stone
8:00am ESTOpening Remarks
Industry updates
8:10amOrchard TherapeuticsJen Pyron
8:40amMirum PharmaceuticalsDr. Rana Dutta
9:00amVigil NeuroscienceDr. Petra Kaufmann
9:20amForge BiologicsDr. Maria Escolar
9:50amClinical translation of a Krabbe Disease gene therapy without HSC transplantDr. Steven Gray
10:40amOverview of Vanishing White MatterDr. Joshua Bonkowsky
11:40amTransition of CareCatie Becker
12:00pmLunchSunrise, 1st Floor HV Lodge
1:00pmPanel Discussion: Consensus on long term follow-up for Leukodystrophy NBS patientsDr. Ali Fatemi, Dr. Joanne Kurtzberg, Dr. James Provenzale, Dr. Margie Ream, Dr. Robert Thompson Stone, Amy WhiteFairway, 2nd Floor HV Lodge
2:00pmLeukodystrophy Focused Payer EducationSarah Waite-Ardini
2:30pmNext steps for LCN Clinical Practice GuidelinesDr. Margie Ream
4:00pmMeeting Closes
6:00pmFamily Welcome DinnerSunrise, 1st Floor HV Lodge
Click here to view Thursday's agenda