Abstract
Data on disease-modifying therapy (DMT) use for patients with spinal muscular atrophy type 2 (SMA2) are limited. We sought to describe real-world outcomes and health care resource utilization (HCRU) for patients in the United States with SMA2 aged ≥6 months at time of treatment with onasemnogene abeparvovec (OA) monotherapy or nusinersen switching to OA.
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