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 ...

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 ...