•Convenient, one-time infusion treatment
•Treats SMA by focusing on the genetic problem
•Help babies achieve certain
milestones, such as sitting by themselves for 30 seconds or more
•Given as an infusion into the vein, so it might be uncomfortable for small children
•Not approved to treat children with advanced SMA, such as complete paralysis of arms and legs, or using a permanent ventilator
•Child must get regular lab work for at least 3 months after treatment