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Improving Diagnosis and Clinical Monitoring of Acid Sphingomyelinase Deficiency

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.
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 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
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