Administration of New Treatments for SMA

At the time the scientific articles were written, Spinraza® was the first, and only, potentially available disease-modifying drug treatment for SMA.

Spinraza® is delivered directly into the Cerebro Spinal Fluid (CSF) which circulates around the spine and brain; this is how Spinraza® directly reaches the part of the central nervous system that is affected by SMA. Doctors access the CSF using a lumbar puncture which is when a needle is inserted in the lower back through the skin into the space between the back bones of the spine (vertebrae). Doctors may use x-ray to locate the best place for the insertion and they will usually use a local anaesthetic such as a ‘numbing cream’, although occasionally a general anaesthetic may be considered necessary. A small amount of CSF is drawn off and then Spinraza® is injected over one to three minutes.

If Spinraza® is an available and appropriate treatment that is agreed between the medical team and the person or the person’s legal guardian, the medical team need to plan carefully how to administer the drug, so that the intervention and care is safe and appropriate to the physical development of the individual.