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Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular
Melissa D Svoboda1, Nancy Kuntz2, Carmen Leon-Astudillo3
1Division of Pediatric Neurology/Neurodevelopment, Department of Pediatrics, CHRISTUS Children's/Baylor College of Medicine, San Antonio, TX, USA. melissa.svoboda@bcm.edu.
Abstract:
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.
Insights
This study found that risdiplam treatment after onasemnogene abeparvovec showed clinical benefits in children with spinal muscular atrophy (SMA), improving muscle movement and function. Risdiplam was well-tolerated, offering a potential therapeutic option for SMA patients previously treated with onasemnogene abeparvovec.
Area of Science:
- Neurology
- Genetics
- Pharmacology
Background:
- Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder causing progressive muscle weakness.
- Approved treatments include onasemnogene abeparvovec (OA) and risdiplam, targeting different aspects of SMA.
- Understanding sequential treatment strategies is crucial for optimizing patient outcomes.
Purpose of the Study:
- To evaluate the clinical benefits and safety of risdiplam treatment following onasemnogene abeparvovec (OA) in pediatric SMA patients.
- To assess changes in motor function, swallowing ability, and respiratory support needs.
- To provide insights into the sequential use of SMA therapies.
Main Methods:
- A multicenter case series design was employed.
- Data were collected on patients with SMA who received risdiplam after OA treatment.
- Clinical assessments focused on motor function, swallowing, and respiratory status.
Main Results:
- All assessed patients demonstrated stable or improved muscle movement after initiating risdiplam.
- Approximately one-third of patients experienced improved swallowing function.
- A significant portion of patients showed decreased reliance on respiratory support.
Conclusions:
- Sequential treatment with risdiplam after onasemnogene abeparvovec appears safe and beneficial for children with SMA.
- Improvements in motor function, swallowing, and respiratory support were observed.
- Further research with larger cohorts is warranted to confirm these findings.
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