Improving Diagnosis and Clinical Monitoring of Acid Sphingomyelinase Deficiency
By Annenberg Center for Health Sciences
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 DeficiencyMay 26, 2022
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04:12
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>
<li>Overview of olipudase alfa</li>
<li>Efficacy and safety of olipudase alfa from phase 2 and 3 trials</li>
</ul>
May 26, 202226:45
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>
<li>Surveillance recommendations for patients with ASMD</li>
<li>Ongoing symptomatic management and support</li>
<li>Role of liver transplantation</li>
</ul>
May 26, 202210:55
Clinical Management and Treatment Monitoring Part 1
<ul>
<li>Initial evaluation and management of ASMD</li>
<li>Clinical trial enrollment</li>
</ul>
<li>Initial evaluation and management of ASMD</li>
<li>Clinical trial enrollment</li>
</ul>
May 26, 202204:12
Guidelines for the Diagnosis of ASMD
<ul>
<li>Clinical presentations of ASMD</li>
<li>Diagnostic algorithms</li>
<li>Enzymatic testing</li>
</ul>
<li>Clinical presentations of ASMD</li>
<li>Diagnostic algorithms</li>
<li>Enzymatic testing</li>
</ul>
May 26, 202211:31
Defining ASMD
<ul>
<li>Pathophysiology of ASMD</li>
<li>ASMD phenotypes</li>
</ul>
<li>Pathophysiology of ASMD</li>
<li>ASMD phenotypes</li>
</ul>
May 26, 202204:13