2026 Hunter's Hope Symposium
Monday, July 13thDRAFT AGENDA
All DayALL TIMES in Eastern Time ZoneArrive/Check InTamarack Lobby
Tuesday, July 14th8AM - 5PM ET
Start
Time		Stop
Time		Topic/EventSpeakerLocation
7:00 AM7:50 AMBreakfastFairway, 2nd Floor, HV Lodge
8:00 AM 8:10 AMWelcome and IntroductionDr. Robert Thompson Stone and Anna Grantham
Session One: Leukodystrophy Newborn Screening Updates and Discussion
8:10 AM8:40 AMUpdates in NBS for ALD, KD, MLD and CTX in U.S.State LabsDr. Joseph Orsini
8:40 AM9:10 AMThe Evolving Story of Mildly Elevated Psychosine in NBSDr. Dieter Matern
9:10 AM9:20 AMGenoKrabbe DatabaseDr. Robert Thompson Stone
9:20 AM10:20 AMPanel Discussion: Newborn Screening for Krabbe DiseaseDr. Joanne Kurtzberg, Dr. Joseph Orsini, Dr. Dieter Matern, Patricia Hunt, Zachary Leeker, Dr. Robert Thompson Stone
10:20 AM10:35 AMBREAK
10:35 AM11:00 AMFollow-up Protocol for Positive MLD NBSDr. Laura Adang
11:00 AM11:50 AMPanel Discussion: Cases in KD, MLD, and ALD NBSDr. Laura Adang, Dr. Florian Eichler, Dr. Margie Ream
11:50 AM12:00 PMFamily Perspective
12:00 PM1:00 PMLUNCHSunrise, 1st Floor HV Lodge
1:05 PM1:20 PMKrabbe Disease: Genotype-Phenotype CorrelationsAmy WhiteFairway, 2nd Floor, HV Lodge
1:20 PM1:35 PMMetachromatic Leukodystrophy: Genotype-Phenotype Correlations Dr. Mike Gelb
1:35 PM1:50 PMResults of a Delphi Study on Cerebrotendinous Xanthomatosis NBS GuidelinesDr. Andrea DeBarber
1:50 PM2:00 PMQ & A SpeakersWhite, Gelb, DeBarber
Session Two: Therapeutics in KD, ALD, & MLD, CTX
2:00 PM2:15 PMUpdates in KD Preclinical TrialsDr. Steven Gray
2:15 PM2:35PMUpdates in KD Clinical Trials and Gene TherapyDr. Joanne Kurtzberg
2:35 PM2:50 PMUpdate in AAV9 Clinical Trial for KDDr. Florian Eichler
2:50 PM3:05 PMBREAK
3:05 PM3:20 PMUpdates in Treatment of MLD with LenmeldyDr. Paul Orchard
3:20 PM3:50 PMUpdates in ALD TherapeuticsDr. Christine Duncan, Dr. Ali Fatemi, Dr. Florian Eichler
3:50 PM4:05 PMCDCA in Pediatric CTXDr. Florian Eichler
4:05 PM4:50 PMPanel Discussion: Q&A on therapeutics for MLD, ALD, KD and CTX
4:50 PMGroup Photo
6:00 PMDINNERSunrise, 1st Floor HV Lodge
Wednesday, July 15th8AM - 5PM ET
TimeLocation
7:00AM8:00 AMBREAKFASTFairway, 2nd Floor, HV Lodge
Session Three: Therapeutics in Canavan, Alexander Disease, Pelizaeus-Merzbacher Disease, Aicardi-Goutieres, and Vanishing White Matter Disease
8:00 AM8:15 AMGene Therapy for Canavan DiseaseDr. Amanda Nagy
8:15 AM8:45 AMAnti-Sense Therapy for Alexander and Pelizaeus-Merzbacher DiseaseDr. Stephanie Keller
8:45 AM9:00 AMTherapeutics in Aicardi-GoutieresDr. Laura Adang
9:00 AM9:15 AMTherapeutics in Vanishing White Matter DiseaseDr. Florian Eichler
9:15 AM9:45 AMPanel Discussion: Q&A on therapeutics for Canavan, Alexander, PMD, AGS
9:45 AM9:55 AMFamily PerspectiveFairway, 2nd Floor, HV Lodge
9:55 AM10:10 AMBREAK
10:10 AM10:35 AMRapid Approvals and Compassionate Use Programs
Session Four: Optimizing Health and Integration of Care
10:35 AM10:50 AMAdult-Onset Leukodystrophies: Krabbe Disease and the Broader SpectrumDr. Yedda Li
10:50 AM11:05 AMAdult leukodystrophy standards of care and clinical trial considerationsDr. Jennifer Orthmann-Murphy
11:05 AM11:20 AMTransition of CareCatie Becker
11:20 AM11:35 AMIncorporating Palliative CareDr. Jennifer Rubin
11:35 AM12:00 PMPanel Discussion
Family Perspective
12: 00 PM1:00 PMLUNCHSunrise, 1st Floor HV Lodge
1:05 PM1:30 PMOptimizing Musculoskeletal Health: Technologies, Medications, and TherapiesDr. Sally EvansFairway, 2nd Floor, HV Lodge
1:30 PM2:35 PMLCN Workgroups
Krabbe NBS Working Session: Data Review and Category Refinement for Infants with Intermediate PSY LevelsDr. Joanne Kurtzberg & Dr. Margie Ream
LCN Working Session: Transition of Care for Individuals with LeukodystrophiesCatie Becker & Dr. Yedda Li
2:35 PM2: 55 PMReports from Groups
2:55 PM3:00 PMClosing RemarksDr. Robert Stone and Anna Grantham
3:00 PMMedical Meeting Ends
3:05 PM5:40 PMKD NBS Workgroup Meeting
6:00PMFamily Welcome DinnerAll our welcome to attendSunrise, 1st Floor HV Lodge