• 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
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    27 min
  • 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 min
  • Clinical Management and Treatment Monitoring Part 1
    May 26 2022
    • Initial evaluation and management of ASMD
    • Clinical trial enrollment
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    4 min
  • Guidelines for the Diagnosis of ASMD
    May 26 2022
    • Clinical presentations of ASMD
    • Diagnostic algorithms
    • Enzymatic testing
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    12 min
  • Defining ASMD
    May 26 2022
    • Pathophysiology of ASMD
    • ASMD phenotypes
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    4 min