Zolgensma is a treatment given by a doctor or nurse experienced in treating SMA. It is injected into a vein in your child’s arm or leg, in a process called intravenous (IV) infusion. This treatment slowly delivers the medicine into the bloodstream and only needs to be given once.

Zolgensma delivers a new, working gene into the body which mimics SMN1, the gene that is reduced, missing or faulty in people with SMA. The gene is delivered to the motor neurons by a viral vector called AAV9, which is a virus that is not harmful or known to make people sick but instead has been modified to be used for transporting. To make the vector, the DNA of the virus is removed so that the new SMN gene can be put inside. 

Once the gene reaches the motor neuron, it helps the body produce enough SMN protein. The increased amount of SMN protein available to motor neurons can help slow or stop the muscle weakness and wasting in people with SMA from getting worse. Given this, it is important treatment is started as early as possible.

Your neurologist will advise the best treatment option for your child with SMA. 

Zolgensma is available on the Pharmaceutical Benefits Scheme (PBS) for children under 9 months of age, symptomatic and diagnosed with SMA Type 1, or children who do not yet have symptoms but do have one, two or three copies of the SMN2 gene.  

If your child has developed an immunity to AAV9, which is naturally found in the environment, they will not benefit from Zolgensma treatment. As such, all children being considered for treatment with Zolgensma will be tested for the AAV9 antibody, and if they show a response, they will be ineligible for this treatment. As AAV9 antibodies decline over time, children under 9 months age can be retested to check eligibility.

Zolgensma is a gene therapy that has been engineered to contain the SMN1 gene. Treatments like Zolgensma can quickly increase the levels of available SMA protein in the body after administration. Some irreversible damage may have already occurred to the motor neurons that have been without enough SMA protein for some time. This may happen even before a baby is born.

The CMI provides important information about using Zolgensma.