The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy ...

SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...

Jerry Mendell carried out the first phase 1 gene therapy clinical trial for spinal muscular atrophy type 1 at the Abigail Wexler Research Institute, leading to Zolgensma (Onasemnogene abeparvovec ...

Novartis’ AveXis unit has said European regulators have approved its Zolgensma (onasemnogene abeparvovec) gene therapy for the rare childhood wasting disease spinal muscular atrophy (SMA), and ...

The gene therapy is developed for children with ... Novartis’s breakthrough therapy Zolgensma (onasemnogene abeparvovec) is ...