From Clues to Care: Improving Recognition and Treatment of Alpha-Mannosidosis

health, fitness, medicine · EN-US

Alpha mannosidosis (AM) is a rare, progressive lysosomal disorder with a broad and heterogeneous clinical phenotype that can evolve across the lifespan. Drawing on the 2024 Global Delphi Consensus Recommendations and other recent evidence, this activity focuses on practical, clinician oriented strategies for baseline and longitudinal assessment, use of enzyme replacement therapy and hematopoietic stem cell transplantation, monitoring treatment response and disease progression, and the coordination of interprofessional, multidisciplinary care essential for patient management.