Monday, July 14th
All DayArrive/Check InTamarack Lobby
Tuesday, July 15thALL TIMES in Eastern Time Zone
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 GranthamFairway, 2nd Floor, HV Lodge
Session One: Newborn Screening Leukodystrophy Updates and DiscussionFairway, 2nd Floor, HV Lodge
8:10 AM8:35 AMUpdates in KD NBSDr. Dieter MaternFairway, 2nd Floor, HV Lodge
8:35 AM8: 50 AMIKD NBS Follow-up RecommendationsDr. Robert Thompson StoneFairway, 2nd Floor, HV Lodge
8:50 AM9:05 AMClinical and Biochemical Analysis of Individuals with 4 Challenging GALC variantsAmy WhiteFairway, 2nd Floor, HV Lodge
9:05 AM9:50 AMCases Discussion: KD Council, Cases, GenotypeModerator: 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 AM10:05 AMBREAKFairway, 2nd Floor, HV Lodge
10:05 AM10:20 AMUpdates in MLD NBSDr. Joseph OrsiniFairway, 2nd Floor, HV Lodge
10:20 AM10:35 AMUpdates in ALD NBSDr. Eric MallackFairway, 2nd Floor, HV Lodge
10:35 AM10:50AMUpdates in CTX NBSDr. Andrea DeBarberFairway, 2nd Floor, HV Lodge
10:50 AM11:50 AMPanel Discussion: State LabsModerator: Dr. Dieter MaternFairway, 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 AM12:00 PMFamily Perspective: Krabbe DiseaseTammy and Michael WilsonFairway, 2nd Floor, HV Lodge
12:00 PM1:00 PMLUNCHSunrise, 1st Floor HV Lodge
1:10 PM1:25 PMMLD: Genotype-Phenotype Correlations and Follow-up StrategiesDr. Laura AdangFairway, 2nd Floor, HV Lodge
1:25 PM1:40 PMNew Technologies for Biochemical Newborn Screening and Biochemical Post-Screening AnalysisDr. Michael GelbFairway, 2nd Floor, HV Lodge
Session 2: Therapeutics in KD, ALD, MLD, & CTXFairway, 2nd Floor, HV Lodge
1:40 PM2:05 PMUpdates in KD pre-clinical studiesDr. Allison BradburyFairway, 2nd Floor, HV Lodge
2:05 PM2:20 PMUpdates in KD Clinical Trials and HSCTDr. Joanne KurtzbergFairway, 2nd Floor, HV Lodge
2:20 PM2:50 PMPanel Discussion: Unified Clinical Trial Design and Outcome Measures in KDModerator: Dr. Robert Thompson-Stone

Dr. Allison Bradbury, Dr. Joanne Kurtzberg, Dr. Deepa Rajan and Dr. Margie Ream
Fairway, 2nd Floor, HV Lodge
2:50 PM3:05 PMBREAK
3:05 PM3:20 PMGene Therapy for MLD: Challenges and OpportunitiesDr. Paul OrchardFairway, 2nd Floor, HV Lodge
3:20 PM3:35 PMGene Therapy for ALD: Challenges and OpportunitiesDr. Christine DuncanFairway, 2nd Floor, HV Lodge
3:35 PM3:50 PMLeriglitazone clinical trial in ALDDr. Ali FatemiFairway, 2nd Floor, HV Lodge
3:50 PM4:05 PMTherapeutics for CTXDr. Andrea DeBarberFairway, 2nd Floor, HV Lodge
4:05 PM4:40 PMPanel Discussion: Q&A on therapeutics for MLD, ALD, CTXModerator: 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 PMGROUP PHOTO
6:00pmDinnerSunrise, 1st Floor HV Lodge
Wednesday, July 16th
TimeTopic/EventLocation
7:00AM8:00 AMBreakfastFairway, 2nd Floor, HV Lodge
Session 3: Therapeutics in Canavan, Alexander, PMD, Aicardi Goutieres, VWM and ALSPFairway, 2nd Floor, HV Lodge
8:05 AM8:20 AMGene Therapy for Canavan DiseaseDr. Amanda NagyFairway, 2nd Floor, HV Lodge
8:20 AM8:45 AMAnti-Sense Therapy for Alexander and Pelizaeus-Merzbacher DiseaseDr. Stephanie KellerFairway, 2nd Floor, HV Lodge
8:45 AM9:00 AMTherapeutics in Aicardi-GoutieresDr. Laura AdangFairway, 2nd Floor, HV Lodge
9:00 AM9:15 AMTherapeutics in Vanishing White Matter DiseaseDr. Joshua BonkowskyFairway, 2nd Floor, HV Lodge
9:15 AM9:30 AMTherapeutics in ALSPDr. Jennifer Orthmann-Murphy
9:30 AM10:00 AMPanel Discussion: Q&A on therapeutics for Canavan, Alexander, PMD, AGS, VWM and ALSPModerator: 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 AM10:15 AMBREAK
10:15 AM10:35 AMPanel: Pharmaceutical Company Q&AModerator: Eric Mallack

Dr. Arun Mistry* and Angela Tom
Fairway, 2nd Floor, HV Lodge
10:35 AM10:45 AMFamily Perspective: MLDChris and Kristal AbnerFairway, 2nd Floor, HV Lodge
Session 4: Leukodystrophy Care Network Initiatives and PrioritiesFairway, 2nd Floor, HV Lodge
10:45 AM10:55 AMLCN Priorities Anna GranthamFairway, 2nd Floor, HV Lodge
10:55 AM11:30 AMMulti-Disciplinary Clinic: How to Make it WorkDr. Ali Fatemi Fairway, 2nd Floor, HV Lodge
11:30 AM12:00 PMPanel Discussion: Q&A Multi-Disciplinary ClinicModerator: Dr. Joanne KurtzbergFairway, 2nd Floor, HV Lodge
12:00 PM1:00 PMLUNCHSunrise, 1st Floor HV Lodge
1:10 PM1:35 PMRemote Assessments, Wearables, and Durable Medical EquipmentDr. Amena Smith FineFairway, 2nd Floor, HV Lodge
1:35 PM2:00 PMCare of Adults with LeukodystrophyDr. Jennifer Orthmann-MurphyFairway, 2nd Floor, HV Lodge
2:00 PM2:25 PMPost-Transplant Follow-up for KD, ALD, MLDDr. Margie ReamFairway, 2nd Floor, HV Lodge
2:25 PM2: 50 PMPanel 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 PM3:00PMFinal RemarksDr. Robert Stone and Anna GranthamFairway, 2nd Floor, HV Lodge
6:00PMFamily Welcome DinnerSunrise, 1st Floor HV Lodge
Thursday's Agenda