Risdiplam treatment following onasemnogene abeparvovec resulted in muscle movement stability or improvement in all children and improved swallowing and decreased respiratory support in about one third.
Does risdiplam treatment following onasemnogene abeparvovec improve clinical outcomes and safety in children with spinal muscular atrophy?
In children with spinal muscular atrophy, sequential treatment with risdiplam following onasemnogene abeparvovec is well tolerated and may offer clinical benefits in muscle movement, swallowing, and respiratory function.
This Summary of Research summarizes a previously published original article, “Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series.” Spinal muscular atrophy (SMA) is a rare genetic disease that causes muscle weakness and is associated with swallowing and breathing difficulties. Risdiplam (EVRYSDI®) and onasemnogene abeparvovec (OA, ZOLGENSMA®) are two medications approved by the US Food and Drug Administration for the treatment of individuals with SMA. This study explored the clinical benefits and safety of using risdiplam after OA in children with SMA. All children whose muscle movement was assessed showed stability or improvement after risdiplam initiation, and around one in three children saw improvements in swallowing and decreased usage of respiratory support. Risdiplam treatment was well tolerated. This study may help to improve understanding of the potential risks and benefits of using risdiplam treatment after OA treatment in children with SMA. Further studies including more children are necessary.
Svoboda et al. (2026) studied Children with spinal muscular atrophy treated with onasemnogene abeparvovec followed by risdiplam. Risdiplam vs. No risdiplam after onasemnogene abeparvovec was evaluated on Muscle movement stability or improvement. Risdiplam treatment following onasemnogene abeparvovec resulted in muscle movement stability or improvement in all children and improved swallowing and decreased respiratory support in about one third.