The 2024 Scientific Symposium begins on Thursday, June 27.
AM SESSIONS LINK IS FOR 8:00 AM – 12:00 PM CST
PM SESSIONS LINK IS FOR 1:00 – 4:30 PM CST
AM SESSIONS: CENTRAL STANDARD TIME
| 08:00 AM – 08:15 AM | Welcome | Dr. Geneviève Bernard, ULF MSAB Chair Ron Chapleau, ULF Board President |
| 08:15 AM – 08:45 AM | Modulating the ISR for the Benefit of VWM | Dr. Truus Abbink Amsterdam University Medical Center |
| 08:45 AM – 09:15 AM | Leukodystrophies: Much More than Just Diseases of Myelin | Dr. Marianna Bugiani Amsterdam University Medical Center |
| 09:15 AM – 09:45 AM | Real-World Data Supporting Use of CDCA in Pregnancy in CTX | Dr. Tzipora Falik-Zaccai Galilee Medical Center, Ban-Ilan University |
| 09:45 AM – 10:00 AM | Break | |
| 10:00 AM – 10:30 AM | ATAD1 Gene Rescue of Zellweger Spectrum Disorders Via Targeting of Shared Common Mechanism |
Dr. Joshua Bonkowsky Utah Health |
| 10:30 AM – 11:00 AM | Functional Characterization of the IFIH1 Mutations in Aicardi-Goutieres Syndrome: Cytosolic RNA Sensors and Novel Molecular Regulators |
Erika Maghraby University Children’s Hospital V. Buzzi |
| 11:00 AM – 11:30 AM | Comprehensive Evaluation of Genotype-Phenotype Relationships in Pol-III Related Disorders. | Mackenzie Michell-Robinson McGill University |
| 11:30 AM – 12:00 PM | Advances in MSD Diagnosis, Phenotype and Treatment | Dr. Laura Adang Children’s Hospital of Philadelphia |
PM SESSIONS: CENTRAL STANDARD TIME
|
01:00 PM – 01:30 PM |
Leukoencephalopathy with Calcifications and Cysts: Natural History and Clinical Management Considerations |
Dr. Jamie Fraser Children’s National Hospital |
| 01:30 PM – 02:00 PM | Natural History and Imaging Biomarkers of Early-Stage Cerebral Adrenoleukodystrophy |
Dr. Erik Mallack Kennedy Krieger Institute |
| 02:00 PM – 02:30 PM | NIH Research and Neurogenetic Disorders | Dr. Walter Koroshetz National Institutes of Health |
| 02:30 PM – 02:45 PM | Break | |
| 02:45 PM – 03:15 PM | Macrophage Transplantation Rescues RNASET2-Deficient Leukodystrophy by Replacing Deficient Microglia in a Zebrafish Model |
Holly A. Rutherford Sheffield University |
| 03:15 PM – 03:45 PM | Advancements of Gene Therapy for Leukodystrophies | Dr. Allison Bradbury Nationwide Children’s Hospital |
| 03:45 PM – 04:15 PM | Can We Achieve Meaningful Gene Correction in the Leukodystrophies? |
Dr. Florian Eichler Massachusetts General Hospital |
| 04:15 PM – 04:30 PM | Conclusion | Dr. Geneviève Bernard, ULF MSAB Chair Ron Chapleau, ULF Board President |