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