Monday, July 14th | ||||
All Day | Arrive/Check In | Tamarack Lobby | ||
Tuesday, July 15th | ALL TIMES in Eastern Time Zone | |||
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 | Fairway, 2nd Floor, HV Lodge |
Session One: Newborn Screening Leukodystrophy Updates and Discussion | Fairway, 2nd Floor, HV Lodge | |||
8:10 AM | 8:35 AM | Updates in KD NBS | Dr. Dieter Matern | Fairway, 2nd Floor, HV Lodge |
8:35 AM | 8: 50 AM | IKD NBS Follow-up Recommendations | Dr. Robert Thompson Stone | Fairway, 2nd Floor, HV Lodge |
8:50 AM | 9:05 AM | Clinical and Biochemical Analysis of Individuals with 4 Challenging GALC variants | Amy White | Fairway, 2nd Floor, HV Lodge |
9:05 AM | 9:50 AM | 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 |
9:50 AM | 10:05 AM | BREAK | Fairway, 2nd Floor, HV Lodge | |
10:05 AM | 10:20 AM | Updates in MLD NBS | Dr. Joseph Orsini | Fairway, 2nd Floor, HV Lodge |
10:20 AM | 10:35 AM | Updates in ALD NBS | Dr. Eric Mallack | Fairway, 2nd Floor, HV Lodge |
10:35 AM | 10:50AM | Updates in CTX NBS | Dr. Andrea DeBarber | Fairway, 2nd Floor, HV Lodge |
10:50 AM | 11:50 AM | Panel Discussion: State Labs | Moderator: Dr. Dieter Matern | Fairway, 2nd Floor, HV Lodge |
Tracey Bishop (CA)*, Angela Wittenauer (GA), Khaja Basheeruddin (IL)*, Barb Lesko (IN), Zac Leeker (KS), Darrin Sevier (KS), Sondra Rosendahl (MN)*, Miriam Schachter (NJ), Joe Orsini (NY), Jamie Mills (NC), Margie Ream (OH), Taraka Donti (PA), Gwendolyn McKee (TN), Nicolas Szabo (UT), Mei Baker (WI) | ||||
11:50 AM | 12:00 PM | Family Perspective: Krabbe Disease | Tammy and Michael Wilson | Fairway, 2nd Floor, HV Lodge |
12:00 PM | 1:00 PM | LUNCH | Sunrise, 1st Floor HV Lodge | |
1:10 PM | 1:25 PM | MLD: Genotype-Phenotype Correlations and Follow-up Strategies | Dr. Laura Adang | Fairway, 2nd Floor, HV Lodge |
1:25 PM | 1:40 PM | New Technologies for Biochemical Newborn Screening and Biochemical Post-Screening Analysis | Dr. Michael Gelb | Fairway, 2nd Floor, HV Lodge |
Session 2: Therapeutics in KD, ALD, MLD, & CTX | Fairway, 2nd Floor, HV Lodge | |||
1:40 PM | 2:05 PM | Updates in KD pre-clinical studies | Dr. Allison Bradbury | Fairway, 2nd Floor, HV Lodge |
2:05 PM | 2:20 PM | Updates in KD Clinical Trials and HSCT | Dr. Joanne Kurtzberg | Fairway, 2nd Floor, HV Lodge |
2:20 PM | 2:50 PM | Panel Discussion: Unified Clinical Trial Design and Outcome Measures in KD | Moderator: Dr. Robert Thompson-Stone Dr. Allison Bradbury, Dr. Joanne Kurtzberg, Dr. Deepa Rajan and Dr. Margie Ream | Fairway, 2nd Floor, HV Lodge |
2:50 PM | 3:05 PM | BREAK | ||
3:05 PM | 3:20 PM | Gene Therapy for MLD: Challenges and Opportunities | Dr. Paul Orchard | Fairway, 2nd Floor, HV Lodge |
3:20 PM | 3:35 PM | Gene Therapy for ALD: Challenges and Opportunities | Dr. Christine Duncan | Fairway, 2nd Floor, HV Lodge |
3:35 PM | 3:50 PM | Leriglitazone clinical trial in ALD | Dr. Ali Fatemi | Fairway, 2nd Floor, HV Lodge |
3:50 PM | 4:05 PM | Therapeutics for CTX | Dr. Andrea DeBarber | Fairway, 2nd Floor, HV Lodge |
4:05 PM | 4:40 PM | Panel Discussion: Q&A on therapeutics for MLD, ALD, CTX | Moderator: Dr. Joshua Bonkowsky Dr. Laura Adang, Dr. Michael Gelb, Dr. Paul Orchard, Dr. Christine Duncan, and Dr. Andrea DeBarber | Fairway, 2nd Floor, HV Lodge |
4:40 PM | GROUP PHOTO | |||
6:00pm | Dinner | Sunrise, 1st Floor HV Lodge | ||
Wednesday, July 16th | ||||
Time | Topic/Event | Location | ||
7:00AM | 8:00 AM | Breakfast | Fairway, 2nd Floor, HV Lodge | |
Session 3: Therapeutics in Canavan, Alexander, PMD, Aicardi Goutieres, VWM and ALSP | Fairway, 2nd Floor, HV Lodge | |||
8:05 AM | 8:20 AM | Gene Therapy for Canavan Disease | Dr. Amanda Nagy | Fairway, 2nd Floor, HV Lodge |
8:20 AM | 8:45 AM | Anti-Sense Therapy for Alexander and Pelizaeus-Merzbacher Disease | Dr. Stephanie Keller | Fairway, 2nd Floor, HV Lodge |
8:45 AM | 9:00 AM | Therapeutics in Aicardi-Goutieres | Dr. Laura Adang | Fairway, 2nd Floor, HV Lodge |
9:00 AM | 9:15 AM | Therapeutics in Vanishing White Matter Disease | Dr. Joshua Bonkowsky | Fairway, 2nd Floor, HV Lodge |
9:15 AM | 9:30 AM | Therapeutics in ALSP | Dr. Jennifer Orthmann-Murphy | |
9:30 AM | 10:00 AM | Panel Discussion: Q&A on therapeutics for Canavan, Alexander, PMD, AGS, VWM and ALSP | Moderator: Dr. Rafael Galindo Dr. Amanda Nagy, Dr. Stephanie Keller, Dr. Laura Adang, Dr. Joshua Bonkowsky, Dr. Jennifer Orthmann-Murphy | Fairway, 2nd Floor, HV Lodge |
10:00 AM | 10:15 AM | BREAK | ||
10:15 AM | 10:35 AM | Panel: Pharmaceutical Company Q&A | Moderator: Eric Mallack Dr. Arun Mistry* and Angela Tom | Fairway, 2nd Floor, HV Lodge |
10:35 AM | 10:45 AM | 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 | |||
10:45 AM | 10:55 AM | LCN Priorities | Anna Grantham | Fairway, 2nd Floor, HV Lodge |
10:55 AM | 11:30 AM | Multi-Disciplinary Clinic: How to Make it Work | Dr. Ali Fatemi | Fairway, 2nd Floor, HV Lodge |
11:30 AM | 12:00 PM | Panel Discussion: Q&A Multi-Disciplinary Clinic | Moderator: Dr. Joanne Kurtzberg | Fairway, 2nd Floor, HV Lodge |
12:00 PM | 1:00 PM | LUNCH | Sunrise, 1st Floor HV Lodge | |
1:10 PM | 1:35 PM | Remote Assessments, Wearables, and Durable Medical Equipment | Dr. Amena Smith Fine | Fairway, 2nd Floor, HV Lodge |
1:35 PM | 2:00 PM | Care of Adults with Leukodystrophy | Dr. Jennifer Orthmann-Murphy | Fairway, 2nd Floor, HV Lodge |
2:00 PM | 2:25 PM | Post-Transplant Follow-up for KD, ALD, MLD | Dr. Margie Ream | Fairway, 2nd Floor, HV Lodge |
2:25 PM | 2: 50 PM | Panel Discussion: Leukodystrophy Care Network | Moderator: Dr. William Benko Dr. Amena Smith Fine, Dr. Jennifer Orthmann-Murphy, and Dr. Margie Ream | Fairway, 2nd Floor, HV Lodge |
2: 50 PM | 3:00PM | Final Remarks | Dr. Robert Stone and Anna Grantham | Fairway, 2nd Floor, HV Lodge |
6:00PM | Family Welcome Dinner | Sunrise, 1st Floor HV Lodge | ||
Thursday's Agenda |