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Spinal Muscular Atrophy is rare neuromuscular disease that affects the physical motion of affected person, impacting the ability to walk, eat and breathe. SMA directly impacts the motor neuron in the spinal cord and wasting of muscle, the age group affected by SMA is from early childhood to adulthood. Universally, the ratio of SMA is 1 in 6000 or 1 in 10,000, categorized as a rare disease, but the mortality rate is very high.Life expectancy is less than two years.Though SMA completely hampers the vital physical motion of the affect individual, it does not have any impact on person’s ability to think, learn, understand or built any relationship which translated that a person is sound by mind.
SMA has wide range of classification, depending upon the beginning of symptoms or severity it has reached in the individual.
SMA is caused by genetic mutation and passed through from parents to childrens. Basically, SMA is caused by mutation in a neuron called Survival Motor Neuron SMN protein -also referred as SMN protein. This SMN protein is critical for the nervous which control our muscles. But with genetic mutation, the physical movement is affected.
In USA, FDA ( Food And Drug Administration) has approved a durg for the treatment of Spinal Muscular Atrophy - SMA, one time gene therapy which would costs whopping US $ 2.1 million.
Such costs of drug will be a huge disadvantage for majority of the parents whose children suffer from SMA type One. Pharmaceuticals companies should strive to bring drug costs within the affordability of majority of people. It is a responsibility of global NGOs, corporations, and global health organisations to make healthcare affordable to every person, primarily to people from less privileged parts of the world.
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