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.
Keywords: Case series; Gene therapy; Onasemnogene abeparvovec; Risdiplam; Spinal muscular atrophy.
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