Ar Rayyan

Researchers are evaluating the effectiveness and safety of risdiplam in young children under 2 years old diagnosed with spinal muscular atrophy (SMA) who have two copies of the SMN2 gene and have previously received gene therapy with onasemnogene abeparvovec but have experienced a plateau or decline in function. This is an open-label, single-arm, multicenter Phase IV study focusing on this specific pediatric population with SMA. The study aims to assess how risdiplam may impact motor development and overall function after gene therapy. Participants will receive risdiplam orally at doses adjusted according to their weight and age. The treatment is given during the study period, with the dose tailored to each child's characteristics. This study does not include a comparator group and monitors the participants over a treatment period of 72 weeks. The intervention involves continuous administration of risdiplam, and the study design allows observation of changes in motor function following prior gene therapy. During the study, children will be closely monitored through various assessments, including the Bayley Scales of Infant and Toddler Development to measure gross motor skills at baseline and after 72 weeks of treatment. Researchers will track changes in motor abilities, swallowing, respiratory function, and other developmental milestones. Safety evaluations and laboratory tests will be conducted throughout the study to ensure participant well-being. The total duration of participation includes treatment and follow-up visits over the 72-week period.