A New Medicine Developed for Spinal Muscular Atrophy

Published By : 17 Sep 2014 | Published By : QYRESEARCH

Spinal Muscular Atrophy is a very common neurodegenerative disease in the US. One in every forty individuals is affected by this disorder which causes muscle weakness over a period of time. A new compound has been developed by the researchers at the University of Missouri. This compound has shown its effectiveness in animal models of this ailment. The patent for this compound for use in the US was launched in April this year. 

Chris Lorson, a researcher at the Bond Life Sciences Center has stated that the strategy that is being utilized in the lab to fight spinal muscular atrophy aims at repressing the repressor. 
This therapeutic compound which is an antisense oligonucleotide alters the expression of the gene that has been affected by this disease. 

The spinal motor neuron-1 gene undergoes mutation in individuals affected by spinal muscular atrophy. As a result, the gene loses its ability to process a key protein that enables the functioning of muscular neurons. The lower parts of the human body get affected first by this ailment before reaching the upper portions like the spine and neck. 

Luckily, there is an identical copy of this gene that exists in the human body which is known as spinal motor neuron-2. The drug developed by Chris Lorson aims at working on this specific gene sequence. This drug enables the spinal motor neuron-2 gene to avoid the defective gene and helps in processing the protein that helps in proper and effective functioning of muscle neurons.

Due to the creation of drugs like the one created by Chris Lorson, the National Institutes of Health has listed spinal muscular atrophy as a neurological ailment closest to finding a cure.
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