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

Podcast door Annenberg Center for Health Sciences

Engels

Technologie en Wetenschap

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

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.

Alle afleveringen

4 afleveringen

Patient-Centered Holistic Care

* Integrated Care Coordination * Consensus Recommendations for Routine Follow-UpCare in AM * Detailed Assessment Recommendations by BodySystem

11 mrt 2026 - 9 min

Standard of Care Treatment and Monitoring for AM

* Enzyme Replacement Therapy (Velmanase Alfa) * Pivotal Trials of Velmanase Alfa in AM * Hematopoietic Stem Cell Transplant * Consensus Recommendations for Monitoring Treatment

11 mrt 2026 - 17 min

Identification and Diagnosis of AM

* Phenotypic Subtypes of AMGuidelines for Genetic Testing * Diagnostic Algorithms for AM * Consensus Recommendations for Testing andBaseline Assessment

11 mrt 2026 - 11 min

Introduction to Alpha-Mannosidosis (AM)

* Description * Epidemiology * Case Vignette

11 mrt 2026 - 4 min

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