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Neurology. Genetics|January 15, 2025
Current Advances and Challenges in Gene Therapies for Neurologic Disorders: A Review for the ClinicianGiulia Stefania Porcari, John Warren Collyer, Laura Ann Adang, et al.Journal of Inherited Metabolic Disease|June 3, 2025
Characterizing Diagnostic Delays in Metachromatic Leukodystrophy: A Real-World Data ApproachAli Mohajer, Anjana Sevagamoorthy, Karen Bean, et al.Molecular Genetics and Metabolism|March 24, 2024
Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approachLaura Ann Adang, Anjana Sevagamoorthy, Omar Sherbini, et al.Neurology|June 27, 2025
Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research DirectionsMarije A B C Asbreuk, Daphne H Schoenmakers, Laura Ann Adang, et al.Molecular Genetics and Metabolism|July 4, 2024
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophyLaura Ann Adang, Samuel Groeschel, Chloe Grzyb, et al.Pageof 1