Report Overview

The UK 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.

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 the 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 the movement of the hands and feet.

SMA types 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 diseases. In the UK approximately 100 children are born with this rare disease each year. Moreover, there are between some 1200 - 2500 children and adults in the UK living with SMA. Approximately one in every 40-60 people is an SMA carrier, which is approximately 1.5 million people in the UK. 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 rare disorders. The rising incidence of spinal muscular atrophy due to the increasing population is one of the key factors for the growth in the SMA treatment market. This growth is attributed to the rapidly developing health infrastructure in the UK, the initiatives are 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.

An increase in R&D investment by major companies will boost the market growth in the forecast period. Granting 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.

Market Restraints

However, the high cost of treatment for spinal muscular atrophy is hindering market growth. Additionally, the lack of experienced professionals and less adoption rate of spinal muscular atrophy treatment options is also restraining the market growth.