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
Claim This Podcastby Annenberg Center for Health Sciences
5 episodes
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Podcast Overview
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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Publishing Since
5/26/2022
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Recent Episodes
May 26, 2022
Emerging Therapies: Efficacy and Safety Data from Phase 3 Trials
<ul> <li>Overview of olipudase alfa</li> <li>Efficacy and safety of olipudase alfa from phase 2 and 3 trials</li> </ul>
May 26, 2022
Clinical Management and Treatment Monitoring Part 2
<ul> <li>Surveillance recommendations for patients with ASMD</li> <li>Ongoing symptomatic management and support</li> <li>Role of liver transplantation</li> </ul>
May 26, 2022
Clinical Management and Treatment Monitoring Part 1
<ul> <li>Initial evaluation and management of ASMD</li> <li>Clinical trial enrollment</li> </ul>
5 total episodes available
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