POSB339 Real-World Adherence of Nusinersen for the Treatment of Spinal Muscular Atrophy (SMA) Using US Large Administrative Claims Dataset

Abstract

Nusinersen, approved for the treatment of spinal muscular atrophy (SMA) across all ages, is administered intrathecally at a dose of 12 mg. In the US label, the approved administration starts with 3 loading doses at 14-day intervals, and a 4th loading dose 30 days thereafter. A maintenance dose is then administered every 4 months. In this study, real-world adherence and treatment patterns of nusinersen using a large US Commercial and Medicaid claims database were examined.