This clinical trial is focused on studying a condition known as Spinal Muscular Atrophy (SMA), a genetic disorder that affects the muscles used for movement. The study is investigating the use of a treatment called risdiplam, which is administered as an oral solution. Risdiplam is being evaluated as an early intervention for children who have already received a gene therapy treatment called onasemnogene abeparvovec. The purpose of the study is to assess how effective and safe risdiplam is when given to pediatric patients with SMA after they have undergone gene therapy.

Participants in the study will receive risdiplam for a period of time, and the effects on their motor skills will be monitored. The study will look at changes in the participants’ ability to move and perform physical activities over the course of the treatment. The treatment is given in a liquid form that can be taken by mouth or through a feeding tube if necessary. The study aims to provide valuable information on how risdiplam can help improve the quality of life for children with SMA.

Throughout the study, researchers will carefully observe the participants to ensure their safety and to gather data on how well the treatment works. The study is designed to last for a specific period, during which the participants’ progress will be regularly assessed. This research is important for understanding how early intervention with risdiplam can benefit children with SMA who have already received gene therapy, potentially leading to better outcomes for those affected by this condition.