Risdiplam add-on therapy following onasemnogene abeparvovec resulted in clinical improvement for most children with spinal muscular atrophy and 2 SMN2 copies.
Does add-on risdiplam improve clinical outcomes in children with spinal muscular atrophy and 2 SMN2 copies previously treated with onasemnogene abeparvovec?
Add-on risdiplam may offer clinical improvement for children with SMA and 2 SMN2 copies who experience clinical deterioration after initial treatment with onasemnogene abeparvovec.
Absolute Event Rate: 0% vs 0%
Our results indicate an improvement in most children in a case series through add-on risdiplam. Evaluating clinical outcome parameters in clinical practice may prove challenging and should be complemented by the parental perspective. The decision regarding the use of add-on therapy in children with SMA who receive one line of treatment but show a clinical deterioration should be considered on an individual level, and assessments of predefined therapeutic goals are recommended.
Stoltenburg et al. (Sat,) reported a other. Risdiplam add-on therapy following onasemnogene abeparvovec resulted in clinical improvement for most children with spinal muscular atrophy and 2 SMN2 copies.