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Myotonic Dystrophy Type 1 (DM1): HARMONIA - A Global Phase 3 Trial Assessing the Efficacy and Safety of Z-basivarsen
Activity Snapshot
12:00 PM EST
Join us for an overview of HARMONIA, a Phase 3 study designed to assess the efficacy and safety of zeleciment basivarsen, an investigational medicine, in individuals living with myotonic dystrophy type 1 (DM1). The discussion will cover the design of the investigational FORCETM platform intended to enable targeted tissue delivery, and an overview of the HARMONIA study including study design, eligibility criteria, the scientific rationale for selecting the 5xSTS as the primary endpoint and the study's secondary and exploratory endpoints spanning multiple organ systems, including the CNS.

