Onasemnogene Abeparvovec: First Global Approval

This article summarizes the milestones in the development of onasemnogene abeparvovec leading to this f irst approval for the treatment of paediatric patients aged < 2 years with SMA and bi-allelic mutations inSMN1.

http://link.springer.com/10.1007/s40265-019-01162-5

Source: Drugs - July 2, 2019 Category: Drugs & Pharmacology Source Type: research