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

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

Defining ASMD

* Pathophysiology of ASMD * ASMD phenotypes