Novartis (United States)

Real-World Treatment Patterns in Patients with Spinal Muscular Atrophy Receiving Multiple Disease-Modifying Therapies

Nusinersen (approved in 2016), onasemnogene abeparvovec (intravenous infusion [OAV], 2019; intrathecal injection, 2025), and risdiplam (2020) are disease-modifying therapies (DMTs) available in the US for spinal muscular atrophy (SMA). Real-world evidence on treatment patterns am...

Aggregation framework for continuous digital health measures and application to walking activity data

Abstract No standardized procedures for aggregating continuous digital health measures exist. Using a modified Delphi approach, we developed a framework to standardize the selection of aggregations that provide clinically meaningful, stable values. This was applied to aggregate s...