Patients with spinal muscular atrophy (SMA) treated with a more expensive medication were found to have higher pharmacy costs but lower SMA-related health care resource utilization and medical costs compared with patients receiving standard-of-care nusinersen monotherapy.
A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1.