5 episodes
Improving Diagnosis and Clinical Monitoring of Acid Sphingomyelinase Deficiency Annenberg Center for Health Sciences
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- 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.
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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 -
Clinical Management and Treatment Monitoring Part 2
Surveillance recommendations for patients with ASMD
Ongoing symptomatic management and support
Role of liver transplantation -
Clinical Management and Treatment Monitoring Part 1
Initial evaluation and management of ASMD
Clinical trial enrollment -
Guidelines for the Diagnosis of ASMD
Clinical presentations of ASMD
Diagnostic algorithms
Enzymatic testing -