NVS - Novartis Reveals Promising Gene Therapy Data for Treating Spinal Muscular Atrophy in Older Children | Benzinga
Monday, Novartis AG (NYSE:NVS) presented new data for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA).
Final data from the SMART Phase 3b study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ? 8.5 kg to ? 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease-modifying therapy at the time of treatment.
The primary study objective was to evaluate the safety and tolerability of Zolgensma in older and heavier patients than were treated in previous clinical studies.
There were no reported cases of ...