Onasemnogene abeparvovec

HOW IT IS BUILT The methods behind it Spinal muscular atrop… Intrathecal administr… WHAT IT CAN DO Its core abilities WHERE IT IS USED — · — — · — OA learns · reasons · acts
How it's built
What it can do
Where it's used

Onasemnogene abeparvovec is a gene therapy medicine designed to treat spinal muscular atrophy (SMA), a rare genetic disorder that causes progressive muscle weakness and paralysis. The drug works by delivering a functional copy of the missing or defective SMN1 gene directly into patients' cells, helping their bodies …