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

PODCAST · health

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.

  1. 4

    Patient-Centered Holistic Care

    Integrated Care CoordinationConsensus Recommendations for Routine Follow-UpCare in AM  Detailed Assessment Recommendations by BodySystem

  2. 3

    Standard of Care Treatment and Monitoring for AM

    Enzyme Replacement Therapy (Velmanase Alfa)Pivotal Trials of Velmanase Alfa in AMHematopoietic Stem Cell TransplantConsensus Recommendations for Monitoring Treatment

  3. 2

    Identification and Diagnosis of AM

    Phenotypic Subtypes of AMGuidelines for Genetic TestingDiagnostic Algorithms for AMConsensus Recommendations for Testing andBaseline Assessment

  4. 1

    Introduction to Alpha-Mannosidosis (AM)

    DescriptionEpidemiologyCase Vignette

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ABOUT THIS SHOW

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.

HOSTED BY

Annenberg Center for Health Sciences

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