The Australian Spinal Muscular Atrophy Therapeutics Market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
The Spinal Muscular Atrophy (SMA) is a group of genetic neuromuscular disorders that affect the nerve cells that control voluntary muscles (motor neurons). The loss of motor neurons causes progressive muscle weakness and loss of movement due to muscle wasting (atrophy). The severity of the symptoms, the age at which symptoms, begin, and genetic cause varies by type. Many types of SMA mainly affect the muscles involved in walking, sitting, arm movement, and head control. Breathing and swallowing may also become difficult as the disease progresses in many types of SMA. In some types of SMA, the loss of motor neurons makes it hard to control movement of the hands and feet.
SMA type 1, 2, 3, and 4 are caused by changes (or pathogenic mutations) in the SMN1 gene and are inherited in an autosomal recessive manner. The disease generally manifests early in life and is the leading genetic cause of death in infants and toddlers. SMA is caused by defects in the Survival Motor Neuron 1 (SMN1) gene that encodes the SMN protein. The SMN protein is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle contraction (motor neurons).
SMA is one of the most common rare disease. Approximately one in 6,000 babies born have SMA, and about one in 40 people carry a copy of the altered gene that causes the condition (although they do not have the condition themselves).
Market Growth Drivers
The overall burden of Spinal Muscular Atrophy is one of the highest in terms of cost and healthcare services in the rare disorder. The rising incidence of Spinal Muscular Atrophy due to the increasing population is one of the key factors for the growth in SMA treatment market. This growth is attributed to the rapidly developing health infrastructure in the Australia, the initiatives undertaken by various public, as well as non-profit, organizations for creating awareness regarding SMA have increased remarkably which is propelling the demand for efficient therapeutic options leading to better patient outcomes.
Increase in R&D investment by major companies will boost the market growth in the forecast period. Granting of various drug designation to novel drugs is also accelerating the market growth for spinal muscular atrophy treatment. In recent times, there have been few substantial and very significant product launches in gene therapy for the treatment of spinal muscular atrophy disease.
However, the high cost of treatment for Spinal Muscular Atrophy is hindering the market growth. Additionally, lack of experienced professionals and less adoption rate of Spinal Muscular Atrophy treatment options are also restraining the market growth.