Improving Diagnosis and Clinical Monitoring of Acid Sphingomyelinase Deficiency

Annenberg Center for Health Sciences
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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.

Episodes

  1. 05/26/2022

    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

  2. 05/26/2022

    Clinical Management and Treatment Monitoring Part 2

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

  3. 05/26/2022

    Clinical Management and Treatment Monitoring Part 1

    Initial evaluation and management of ASMD Clinical trial enrollment

  4. 05/26/2022

    Guidelines for the Diagnosis of ASMD

    Clinical presentations of ASMD Diagnostic algorithms Enzymatic testing

  5. 05/26/2022

    Defining ASMD

    Pathophysiology of ASMD ASMD phenotypes

  • 5 Episodes

About

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.

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