New study expands evaluation of gene therapy for spinal muscular atrophy German researchers have just published results of a retrospective analysis of routine data collected from all patients with spinal muscular atrophy (SMA) treated with Zolgensma® (onasemnogene abeparvovec - adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy…..
Trend Chart On Innovative Bioindustries - June 2020, 2nd FEATURE STORY ● Novartis snags EU nod for SMA gene therapy After a manufacturing-related delay, Novartis has won conditional approval in the EU for Zolgensma®, the one-time gene therapy to treat patients with a clinical diagnosis of SMA type 1 and…..
