Nusinersen in adults with type 3 spinal muscular atrophy: long-term outcomes on motor and respiratory function

Nusinersen was approved for 5q spinal muscular atrophy (SMA), irrespective of age, SMA type or functional status. Nonetheless, long-term data on adults with milder phenotypes are scarce. We aimed to characterize evolution on motor and respiratory function in our cohort of adults with type 3 SMA. We conducted a longitudinal retrospective single-center study, including adults (≥18 years) with type 3 SMA under nusinersen for > 22 months. We reported on motor scores and spirometry parameters. Ten patients were included, with a median follow-up of 34 months (range = 22–46). Four patients (40