5 episodes

Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disorder that causes accumulation of sphingomyelin in various organs. In addition to a low incidence, ASMD has a heterogeneous presentation that makes management challenging. Join experts Margaret McGovern, MD, PhD, and Pramod Mistry, MD, PhD, in this review of the diagnosis and treatment of patients with ASMD.

Improving Diagnosis and Clinical Monitoring of Acid Sphingomyelinase Deficiency Annenberg Center for Health Sciences

    • Health & Fitness

Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disorder that causes accumulation of sphingomyelin in various organs. In addition to a low incidence, ASMD has a heterogeneous presentation that makes management challenging. Join experts Margaret McGovern, MD, PhD, and Pramod Mistry, MD, PhD, in this review of the diagnosis and treatment of patients with ASMD.

    Emerging Therapies: Efficacy and Safety Data from Phase 3 Trials

    Emerging Therapies: Efficacy and Safety Data from Phase 3 Trials

    Overview of olipudase alfa
    Efficacy and safety of olipudase alfa from phase 2 and 3 trials

    • 26 min
    Clinical Management and Treatment Monitoring Part 2

    Clinical Management and Treatment Monitoring Part 2

    Surveillance recommendations for patients with ASMD
    Ongoing symptomatic management and support
    Role of liver transplantation

    • 10 min
    Clinical Management and Treatment Monitoring Part 1

    Clinical Management and Treatment Monitoring Part 1

    Initial evaluation and management of ASMD
    Clinical trial enrollment

    • 4 min
    Guidelines for the Diagnosis of ASMD

    Guidelines for the Diagnosis of ASMD

    Clinical presentations of ASMD
    Diagnostic algorithms
    Enzymatic testing

    • 11 min
    Defining ASMD

    Defining ASMD

    Pathophysiology of ASMD
    ASMD phenotypes

    • 4 min

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