Patients with spinal muscular atrophy (SMA) treated with risdiplam use inpatient service less, stay in hospital shorter, and have fewer hospital visits and SMA-related complications, according to results presented at the 2023 Muscular Dystrophy Association Clinical & Scientific Conference. This is the case even for patients who were previously treated with nusinersen. 

“Future analyses are needed to assess risdiplam’s impact on healthcare costs and other SMA-relevant outcomes,” the study authors wrote. They added that these results should also be compared with other SMA treatments and patients who received no treatment.

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The use of healthcare resources and disease-related complications were substantial among patients with SMA before the approval of disease-modifying therapies such as risdiplam and nusinersen, the researchers noted.

In the present study, a team of researchers led by Elmor Pineda, PharmD, RPh, MS, from Genentech in San Francisco, California, explored the healthcare resource usage and SMA-related complications among patients treated with risdiplam. 

The researchers analyzed 63 patients with SMA, who were followed up for a mean period of 9.9 months. Mean age was 20 years. Of these 63 patients, the majority (70%) were previously treated with nusinersen. 

They found that there was an overall reduction in complications related to the disease among patients treated with risdiplam, including shortness of breath, acute and chronic respiratory failure, and pneumonia, as well as orthopedic and voice, and speech impediments. This was the case whether or not patients were previously treated with nusinersen. 

Most patients (86%) had no hospitalizations before or after the first risdiplam claim. Among those hospitalized, the mean number of hospitalizations increased from 1.44 before the first claim to 1.99 after. On the contrary, the mean length of hospital stay and the number of inpatient services decreased, as did the mean number of outpatient visits.

Risdiplam marketed under the brand name Evrysdi® is the latest disease-modifying therapy approved by the US Food and Drug Administration for the treatment of SMA. It works by increasing the amount of functional SMN protein produced by the SMN2 gene.


Shieh P, Geffen D, Boudreau D, et al. A retrospective analysis of healthcare resource utilization (HCRU) and disease-related complications among risdiplam-treated patients with SMA. Poster presentation at: 2023 Muscular Dystrophy Association Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX.