There are two pharmaceutical treatments now available for people with SMA.
Nusinersen (Spinraza) is approved by the Food and Drug Administration (FDA) for use in children and adults. The medication is injected into the fluid surrounding the spinal cord. It improves head control and the ability to crawl or walk, among other mobility milestones in infants and others with certain types of SMA.
The other FDA-approved treatment is onasemnogene abeparvovec (Zolgensma). It’s intended for children under 2 years old with the most common types of SMA.
An intravenous medication, it works by delivering a functional copy of an SMN1 gene into the child’s target motor neuron cells. This leads to better muscle function and mobility.
The first four doses of Spinraza are administered over a period of 72 days. Afterward, maintenance doses of the medication are administered every four months. Children on Zolgensma receive a one-time dose of the medication.
Talk with your child’s doctor to determine whether either medication is right for them. Other treatments and therapies that may bring relief from SMA include muscle relaxers and mechanical, or assisted, ventilation.