Meeting Agenda*
Wednesday Evening (March 18, 2026)
- Welcome Reception (6:00 PM - 6:45 PM)
- Scientific Session #1 - Treatment (6:45 PM - 8:15 PM)
- Intrathecal Delivery of GALC Via Extracellular Vesicles as a Transient Immunomodulatory Therapy in Krabbe Disease (Maria Irene Givogri)
- Challenging the Capacity of AAV-GALC Gene Therapy to Cope with Modifiers of Adult Brain Function (Ernesto Bongarzone)
- The Preconditioning Regimen for Hematopoietic Stem Cell Transplantation Causes Ablation of Neurogenesis in the Dentate Gyrus of the Hippocampus (Steven LeVine, Scott Sands)
Thursday (March 19, 2026)
-
Breakfast (7:30 AM - 8:00 AM)
-
Welcome & Introductions (8:00 AM)
- Scientific Session #2 - Pathology (8:15 AM - 10:15 AM):
- Impaired Docking and Recycling of Synaptic Vesicles in Krabbe Disease (Diego Zelada)
- Cell-Specific Roles of GALC in Motor Learning–Induced Adaptive Myelination (Natalia Saldivia)
- Investigating the Immune Landscape in Krabbe Disease (Anthony J. Filiano)
- Untargeted metabolomics analysis of Krabbe Diease Biospecimens Uncovers Putative Novel Biomarkers (Rachel Wurth)
- GenoKrabbe: A comprehensive Genotype-Phenotype Database for Krabbe Disease (Robert Thompson Stone)
- Coffee break
- Scientific Session #3 - Newborn Screening (10:30 AM - 12:30 PM):
- Krabbe Disease Newborn Screening in Wisconsin and Beyond: U.S. State Laboratory Experiences Following RUSP Inclusion (Part 1 - Amy White)
- Krabbe Disease Newborn Screening in Wisconsin and Beyond: U.S. State Laboratory Experiences Following RUSP Inclusion (Part 2 - Kaegan Mestel)
- Krabbe Disease Newborn Screening in Wisconsin and Beyond: U.S. State Laboratory Experiences Following RUSP Inclusion (Part 3 - Zoe Culshaw-Klein)
- Late Infantile Krabbe Disease: Something worth Screening For! (Amy White)
- Long-term Follow-Up Roadmap for Newborn Screening (Marci Sontag)
- Lunch break
- Session #4 - Roundtable Workshop I (1:45 PM - 5:15 PM)
* Concurrent workshops will meet for 2 hours, followed by a 30 minute readout from each roundtable to the general audience- Basic Science - this roundtable will consider pathogenicity (including immunology) from conception to adulthood, and potential optimized disease models: KD animal models and alternatives (for all KD subtypes)
- Translational Science - this roundtable will consider therapeutic alternatives beyond stem cell transplantation incluing but not limited to gene therapy, large and small molecules, etc. as either therapeutic alternatives, a bridge to transplant, or therapeutic adjuncts (for all KD subtypes)
- Clinical - this roundtable will consider open access, centralized, natural history database and biorepository, biomarkers, severity stratification of GALC variants, gray zone characterization, prognostic, predictive, and surrogate biomarkers, therapeutic safety and efficacy outcomes (for all KD subtypes)
- Dinner & Networking Opportunity (6:15 PM)
Friday Morning (March 20, 2026)
- Breakfast (7:30 AM - 8:00 AM)
- Welcome & Sue Rosenau Legacy Award presentation (8:00 AM)
- Session #5 - Roundtable Workshop II (8:30 AM - 11:30 AM)
* Concurrent workshops will meet for 2 hours, followed by a 30 minute readout from each roundtable to the general audience- Translational Science - this roundtable will consider the application of genomics, epigenomics, proteomics, metabolomics, single cell and spatial biology, to enhance disease understanding and treatment response, identify potential prognostic, predictive, and surrogate biomarkers, improve the characterization of GALC variants through optimized functional assays or via alternative approaches, identify and characterize pathway genes, and further refine NBS (for all KD subtypes)
- Clinical - this roundtable will consider how to optimize, standardize, and deliver consistent, information, treatments, interventions, and clinical management beginning with NBS across all geographies and centers (for all KD subtypes)
- KrabbeConnect Family Panel (11:30 AM - 12:30 PM)
- Lunch and departures (12:30 PM - 1:30 PM)
* participation in KrabbeConnect event