Objective(s): 1 Explain how pathophysiology informs trial design for the leukodystrophies. 2 Discuss the different gene therapy approaches for the leukodystrophies. 3 Describe biomarkers monitoring and management of leukodystrophy trials.Location: Nationwide Children’s Hospital Conference CenterSpecialties: General PediatricsFaculty Disclosures: Florian Eichler, MD (Consulting Fee-Atlas Venture|Consulting Fee-ASPA Therapeutucs|Consulting Fee-Acadia|Consulting Fee-Leal Therapeutics|Consulting Fee-Orchard Therapeutics|Consulting Fee-Sanofi Therapeutics|Consulting Fee-Spoila Therapeutics|Consulting Fee-Spur Therapeutics|Consulting Fee-Takeda Therapeutics|Consulting Fee-Up to Date|Grant or research support-GLIA CTN|Grant or research support-European Leukodystrophy Associateion|Ownership-Swan Bio – 03/21/2025)Download Handout
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2027 Pediatric Dermatology Journal Club
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ReleasedMar 27, 2025
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Last day to claim creditMar 28, 2028 1 year left
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American Society of Neuroradiology Education Advances: Pediatric Leukodystrophies: Practical Points through a Case Review
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American Society of Neuroradiology Spotlight on Leukodystrophies (LDs) and Inborn Errors of Metabolism (IEMs)
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PRIME Education, Inc. (PRIME®) The Paradigm Shift in Neuro-Oncology: Precision Decision-Making for IDH1/2 Mutated Gliomas
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Nationwide Children’s Hospital 2025 PGROD: Cell Therapy for Cancer and Beyond by Dean Lee, MD, PhD
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Nationwide Children’s Hospital 2025 PGROD – The Global One Health Paradigm: Addressing Shared Health Challenges through Collaboration
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Nationwide Children’s Hospital 2025 PGROD: Building Capacity to Assess Mediators of Outcomes and Inequities: A Story of Collaboration by Brett Anderson, MD, MBA, MS
