Spinal muscular atrophy is a hereditary neurological disorder characterized by loss of motor neurons innervating different muscles of the body. Despite being a rare disorder, affecting around one infant out of at least 10,000 live births, it is still one of the leading hereditary causes of infant disability and death. This disorder has a wide range of subtypes and symptom severity.

The most common SMA, which is known as type 1 SMA, accounts for more than half of the cases and it is a severe form of SMA that follows an autosomal recessive inheritance. Spinal muscle atrophy mainly occurs due to a genetic abnormality in the SMN gene coding for proteins responsible for normal nerve functioning. This protein abnormality affects motor neurons – i.e. nerves supplying muscles versus sensory nerves – therefore causing characteristics of SMA (1-3).