| 2025 Hunter's Hope Medical Symposium DRAFT Agenda | ||||||
| All times are Eastern Time | Subject to Change | |||||
| DRAFT AGENDA | ||||||
| Monday, July 14th | ||||||
| Time | Topic/Event | Location | ||||
| All Day | Arrive/Check In | Tamarack Lobby | ||||
| Tuesday, July 15th | ||||||
| Time | Topic/Event | Speaker | Location | |||
| 8AM - 5PM EST | Medical Symposium, Day One | |||||
| 7:00AM | Breakfast | Fairway, 2nd Floor, HV Lodge | ||||
| 8:00AM | Welcome and Introduction | Dr. Robert Thompson Stone and Anna Grantham | Fairway, 2nd Floor, HV Lodge | |||
| Session One: Newborn Screening Leukodystrophy Updates and Discussion | ||||||
| Updates in KD NBS | Dr. Dieter Matern | |||||
| IKD NBS Follow-up Recommendations | Dr. Robert Thompson Stone | |||||
| Clinical and Biochemical Analysis of Individuals with 4 Challenging GALC variants | Amy White | |||||
| Cases Discussion: KD Council, Cases, Genotype | Moderator: Dr. Joanne Kurtzberg Panelists: Dr. Dieter Matern, Dr. Joe Orsini, Dr. Margie Ream, Dr. Robert Thompson Stone, Amy White | |||||
| BREAK | ||||||
| Updates in MLD NBS | Dr. Joseph Orsini | |||||
| Updates in ALD NBS | Dr. Eric Mallack | |||||
| Updates in CTX NBS | Dr. Andrea DeBarber | |||||
| Panel Discussion: State Labs | Moderator: Dr. Dieter Matern | |||||
| Family Perspective: Krabbe Disease | Tammy and Michael Wilson | |||||
| LUNCH | Sunrise, 1st Floor HV Lodge | |||||
| Session 2: Therapeutics in KD, ALD, MLD, & CTX | ||||||
| Updates in KD pre-clinical studies | Dr. Allison Bradbury | |||||
| Updates in KD Clinical Trials and HSCT | Dr. Joanne Kurtzberg | |||||
| Panel Discussion: Unified Clinical Trial Design and Outcome Measures in KD | ||||||
| Introduction to Gemma Bio | ||||||
| BREAK | ||||||
| MLD: Genotype-Phenotype Correlations and Follow-up Strategies | Dr. Laura Adang | |||||
| Gene Therapy for MLD: Challenges and Opportunities | ||||||
| Gene Therapy for ALD: Challenges and Opportunities | Dr. Christine Duncan | |||||
| Therapeutics for CTX | Dr. Andrea DeBarber | |||||
| Panel Discussion: Q&A on therapeutics for MLD, ALD, CTX | ||||||
| 6:00pm | Dinner | Sunrise, 1st Floor HV Lodge | ||||
| Wednesday, July 16th | ||||||
| Time | Topic/Event | Location | ||||
| 7:30AM - 4:30PM | Medical Symposium, Day Two | |||||
| 7:00AM | Breakfast | Fairway, 2nd Floor, HV Lodge | ||||
| Session 3: Therapeutics in Canavan, Alexander, PMD, and Aicardi Goutieres | ||||||
| 7:30AM | Gene Therapy for Canavan Disease | Dr. Amanda Nagy | Fairway, 2nd Floor, HV Lodge | |||
| Anti-Sense Therapy for Alexander and Pelizaeus-Merzbacher Disease | ||||||
| Therapeutics in Aicardi-Goutieres | Dr. Laura Adang | |||||
| Therapeutics in Vanishing White Matter Disease | Dr. Joshua Bonkowsky | |||||
| Panel Discussion: Q&A on therapeutics for Canavan, Alexander, PMD, AGS, VWM | ||||||
| Panel: Pharmaceutical Company Q&A | ||||||
| BREAK | ||||||
| Family Perspective: MLD | Chris and Kristal Abner | |||||
| Session 4: Leukodystrophy Care Network Initiatives and Priorities | ||||||
| LCN Priorities | Anna Grantham | |||||
| Multi-Disciplinary Clinic: How to Make it Work | Dr. Ali Fatemi | |||||
| Panel Discussion: Q&A Multi-Disciplinary Clinic | Moderator: Dr. Joanne Kurtzberg | |||||
| LUNCH | ||||||
| Remote Assessments, Wearables, and Durable Medical Equipment | Dr. Amena Smith Fine | |||||
| Care of Adults with Leukodystrophy | ||||||
| Post-Transplant Follow-up for KD, ALD, MLD | Dr. Margie Ream | |||||
| Panel Discussion: Leukodystrophy Care Network | Moderator: Joanne Kurtzberg | |||||
| Final Remarks | Dr. Robert Stone and Anna Grantham | |||||
| 4:30PM | Meeting Closes | |||||
| 6:00PM | Family Welcome Dinner | Sunrise, 1st Floor HV Lodge | ||||
