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Improving Diagnosis and Clinical Monitoring of Acid Sphingomyelinase Deficiency

De: Annenberg Center for Health Sciences
  • Resumen

  • 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.
    Annenberg Center for Health Sciences
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Episodios
  • Emerging Therapies: Efficacy and Safety Data from Phase 3 Trials
    May 26 2022
    • Overview of olipudase alfa
    • Efficacy and safety of olipudase alfa from phase 2 and 3 trials
    Más Menos
    27 m
  • Clinical Management and Treatment Monitoring Part 2
    May 26 2022
    • Surveillance recommendations for patients with ASMD
    • Ongoing symptomatic management and support
    • Role of liver transplantation
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    11 m
  • Clinical Management and Treatment Monitoring Part 1
    May 26 2022
    • Initial evaluation and management of ASMD
    • Clinical trial enrollment
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    4 m

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