onasemnogene abeparvovec (Zolgensma)

Onasemnogene abeparvovec (Zolgensma) is a gene therapy designed to treat spinal muscular atrophy (SMA), a genetic disorder that causes muscle weakness and paralysis. It works by delivering a healthy copy of the faulty gene responsible for SMA directly into a child's cells, helping them produce the essential protein they need for muscle function. This treatment aims to stop or slow the disease progression, especially when given early. Zolgensma is administered as a one-time infusion and can offer significant improvements in a child's ability to move and breathe, altering the course of SMA when started promptly.