2026 Hunter's Hope Symposium
Monday, July 13th		DRAFT AGENDA
All Day	ALL TIMES in Eastern Time Zone	Arrive/Check In	Tamarack Lobby
Tuesday, July 14th	8AM - 5PM ET
Start Time	Stop Time	Topic/Event	Speaker	Location
7:00 AM	7:50 AM	Breakfast		Fairway, 2nd Floor, HV Lodge
8:00 AM	8:10 AM	Welcome and Introduction	Dr. Robert Thompson Stone and Anna Grantham
		Session One: Leukodystrophy Newborn Screening Updates and Discussion
		Updates in NBS for ALD, KD, MLD and CTX in U.S.State Labs	Dr. Joseph Orsini
		The Evolving Story of Mildly Elevated Psychosine in NBS	Dr. Dieter Matern
		Panel Discussion: Newborn Screening for Krabbe Disease
		BREAK
		Follow-up Protocol for Positive MLD NBS	Dr. Laura Adang
		Panel Discussion: Cases in KD, MLD, and ALD NBS	Dr. Laura Adang
		Family Perspective
12:00 PM	1:00 PM	LUNCH		Sunrise, 1st Floor HV Lodge
		Krabbe Disease: Genotype-Phenotype Correlations		Fairway, 2nd Floor, HV Lodge
		Metachromatic Leukodystrophy: Genotype-Phenotype Correlations	Dr. Mike Gelb
		Results of a Delphi Study on Cerebrotendinous Xanthomatosis NBS Guidelines
		Session Two: Therapeutics in KD, ALD, & MLD, CTX
		Updates in KD Preclinical Trials	Dr. Steven Gray
		Updates in KD Clinical Trials and Gene Therapy	Dr. Joanne Kurtzberg
		Updates in Treatment of MLD with Lenmeldy	Dr. Paul Orchard
		BREAK
		Updates in ALD Therapeutics	Dr. Christine Duncan, Dr. Ali Fatemi, Dr. Florian Eichler
		CDCA in Pediatric CTX	Dr. Florian Eichler
		Panel Discussion: Q&A on therapeutics for MLD, ALD, KD and CTX
		Group Photo
6:00pm		DINNER		Sunrise, 1st Floor HV Lodge
Wednesday, July 15th	8AM - 5PM ET
Time				Location
7:00AM	8:00 AM	BREAKFAST		Fairway, 2nd Floor, HV Lodge
		Session Three: Therapeutics in Canavan, Alexander Disease, Pelizaeus-Merzbacher Disease, Aicardi-Goutieres, and Vanishing White Matter Disease
		Gene Therapy for Canavan Disease	Dr. Amanda Nagy
		Anti-Sense Therapy for Alexander and Pelizaeus-Merzbacher Disease	Dr. Stephanie Keller
		Therapeutics in Aicardi-Goutieres	Dr. Laura Adang
		Therapeutics in Vanishing White Matter Disease	Dr. Florian Eichler
		Panel Discussion: Q&A on therapeutics for Canavan, Alexander, PMD, AGS
		Family Perspective		Fairway, 2nd Floor, HV Lodge
		BREAK
		Rapid Approvals and Compassionate Use Programs
		Session Four: Optimizing Health and Integration of Care
		Integrating Peds and Adults	Dr. Yedda Li and Dr. Jennifer Orthmann-Murphy
		Transition of Care	Catie Becker
		Incorporating Palliative Care	Dr. Jennifer Rubin
		Panel Discussion
		Family Perspective
11:50 AM	12:50 PM	LUNCH		Sunrise, 1st Floor HV Lodge
		Optimizing Musculoskeletal Health: Technologies, Medications, and Therapies	Dr. Sally Evans	Fairway, 2nd Floor, HV Lodge
		LCN Workgroups
		Krabbe NBS Working Session: Data Review and Category Refinement for Infants with Intermediate PSY Levels	Dr. Joanne Kurtzberg & Dr. Margie Ream
		LCN Working Session: Transition of Care for Individuals with Leukodystrophies	Catie Becker & Dr. Yedda Li
		Reports from Groups
		Closing Remarks	Dr. Robert Stone and Anna Grantham
6:00PM		Family Welcome Dinner	All our welcome to attend	Sunrise, 1st Floor HV Lodge