Key takeaways
Zolgensma (onasemnogene abeparvovec) and Spinraza (nusinersen) are both used to treat spinal muscular atrophy (SMA), but they work in different ways and have different treatment schedules. Zolgensma is a gene therapy given as a one-time IV infusion, specifically for children under 2 years old. It delivers working copies of the SMN1 gene to help the body produce the necessary proteins for muscle movement. In contrast, Spinraza is an antisense oligonucleotide that helps the body make more SMN proteins and is used for both children and adults. Spinraza is injected into the spinal canal and requires multiple doses: four initial loading doses followed by maintenance doses every four months. Zolgensma requires close monitoring for three months post-infusion due to risks like liver problems, while Spinraza requires blood and urine tests before each dose to monitor for bleeding and kidney damage.
