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

Annenberg Center for Health Sciences
  • 0.0 (0)
  • Medicine

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.

Episodes

  1. Mar 11

    Patient-Centered Holistic Care

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

  2. Mar 11

    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. Mar 11

    Identification and Diagnosis of AM

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

  4. Mar 11

    Introduction to Alpha-Mannosidosis (AM)

    DescriptionEpidemiologyCase Vignette

  • 4 Episodes

About

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.

Information