Swiss pharmaceutical giant Novartis (NYSE: NVS) has presented Phase III data this week, demonstrating that the clinical benefits of its gene therapy Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) extend beyond patients up to 2 years of age. The new study has shown similar benefits in older children, including those who have received prior treatment. The trial focused on the safety, tolerability, and efficacy of a one-time infusion of Zolgensma in children aged between 9 months and 9 years, both without disease-modifying therapy and after previous treatment.
The results indicated that the majority of participants maintained their baseline motor milestones, with improvements observed in upper limb function and physical abilities at the 52-week mark. In terms of safety, the study found that most subjects experienced manageable and asymptomatic cases of increased transaminases and transient thrombocytopenia.- Flcube.com