Spinal Muscular Atrophy
SPINRAZA™ (nusinersen (as heptadecasodium) 12mg/5mL solution for injection)
TGA approved in November 2017, SPINRAZA is the first treatment registered in Australia for the treatment of spinal muscular atrophy (SMA).
SPINRAZA is a type of medicine called an anti-sense oligonucleotide (ASO), that works by causing the body to produce more SMN protein.
SPINRAZA is approved for the treatment of 5q SMA.
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Spinal Muscular Atrophy (SMA) is a genetic disease caused by a shortage of a protein called survival motor neuron (SMN). This results in the loss of nerve cells in the spine, leading to weakness of the muscles in the shoulders, hips, thighs and upper back. It may also weaken the muscles used for breathing and swallowing. SMA occurs when a faulty SMN1 gene is passed on to a child by both parents. The SMN1 gene is responsible for the body’s production of SMN protein, which is vital to the function of muscles that enable movement, speech, breathing and swallowing. Without ample production of SMN protein, the nerves of the spinal cord deteriorate and muscle wasting gradually occurs. 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases.
There are four known forms of SMA – Type I, Type II, Type III and Type IV – each determined by the age of onset (from those diagnosed before six months of age to those diagnosed in adulthood) and the physical milestones achieved.