The Efficacy of Risdiplam in 7 Patients Previously Treated With Nusinersen for Spinal Muscular Atrophy

The disease-modifying therapies (DMTs) for spinal muscular atrophy (SMA) produced impressive improvements in motor function and survival in clinical trials. However, there are limited real-world data regarding combination or sequential treatment with DMTs. We report the clinical outcomes of risdiplam in 7 SMA patients previously treated with nusinersen. Treatment effects were assessed based on motor function tests, weight gain, and sensitivity of communication devices. Risdiplam was administered for a median of 30.2 months. The patients’ motor function test scores stabilized. The body weight gain by more than 5% was seen in 3 patients, and the required communication device sensitivity level improved in 1 patient, which had not been observed during nusinersen treatment. The primary reason for switching DMTs was the pain during needle insertion. Sequential treatment of risdiplam had positive effects for motor function and weight gain. Our results may provide useful information when considering a treatment switch.