Spinal muscular atrophy is a rare inherited genetic disease that is characterized by loss of nerve cells, known as motor neurons. Motor neurons receive the nerve impulses, which are transmitted from the brain to the spinal cord and, further transmits these impulses to the muscles with the help of peripheral nerves. The loss of motor neurons can lead to atrophy and weakness in the muscles, that are closest to the trunk of the body such as back, hips and shoulders. Spinal muscular atrophy is mainly caused due to the mutation in the SMA2 and SMN1 genes in chromosome. Any mutation in SMA1 gene can lead to spinal muscular atrophy.
According to National Organization for Rare Disorders, around 95% to 98% people who suffer from spinal muscular atrophy have deletions in the SMA1 (Survival of Motor Neuron 1) gene that results in a decreased production of SMN protein. The prevalence of spinal muscular atrophy has increased in the past few years, due to various risk factors such as smoking, family history, lack of awareness and delay in diagnosis. According to National Organization for Rare Disorders, the incidence of spinal muscular atrophy is approximately one in 10,000 live births. The disease is categorized into four types namely, Type I, Type II, Type III and Type IV. SMA Type I occurs mainly in infants aged between three to six months, with symptoms including very little muscle tone, weak muscles and feeding and breathing problems. SMA Type II and III both are very severe type of spinal muscular atrophy, and affects infants aged between six months to two years. SMA Type IV mainly affects the adult population. Some of the symptoms associated with spinal muscular atrophy include weakness in muscles, breathing difficulty, lack of head control, feeding difficulty and floppy infant (poor muscle tone).
As of June 2017, the spinal muscular atrophy pipeline comprises approximately 16 active drug candidates in different stages of development.
SPINAL MUSCULAR ATROPHY DRUG CANDIDATES UNDER DEVELOPMENT (2017)
Some of the other key players developing drugs for the treatment of spinal muscular atrophy include Cytokinetics, Inc., F. Hoffman La-Roche Ltd, WAVE Life Sciences Ltd. and others.
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