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29 May 2021

Paralysis (also known as Plegia) is a loss of motor functions in one or more muscles. Paralysis can be accompanied by a loss of feeling (sensory loss) in the affected area if there is sensory damage as well as motor damage. It is often caused due to the damage in the nervous system, especially the spinal cord. Muscular Paralysis is a condition where loss of strength in and control over a muscle or group of muscles in a part of the body.

There were many genetic diseases on which several scientists were currently doing on research to discover a cure for that particular genetic disease but in order to find a best medicine for the treatment of the genetic disease, it shall take a lot of time due to several factors. Myotubular Myopathy is a severe genetic disease that leads to muscular paralysis from birth and results in death before two years of age. Currently, the research is going on for the treatment of this genetic disease at the University of Geneva, Switzerland which in working in collaboration with University of Strasbourg, France.

Ninety percent of the affected babies do not live to two years of age. The disease affects the X Chromosome in one in 50,000 male infants. So, only boys are mainly struck by Myotubular Myopathy, since the 2nd X Chromosome in girls generally compensates possible mutation of the first. At present, there is no treatment available for curing this rare genetic disease condition. In general, it shall take several years of period in order to find a gene therapy for genetic disease condition as it involves a set of complicated research studies.

This genetic disease is a life-threatening condition especially for the male infants. This situation has lead the scientists to find a quicker way to counter this genetic disease, so the researchers have turned to a drug molecule which is already been authorised for other treatments in the humans. After a brief study, the scientists have found out that the drug tamoxifen which has been used for many years to treat breast cancer.

This drug molecule has several characteristic properties for protecting the muscle fibers. It is an anti-oxidant, anti-fibrotic and also protects the mitochondria. According to the research studies, it has been proved that the tamoxifen drug has already been used aganist Duchenne Muscular dystrophy which is also an inherited muscular disease that affects only one in 3,500 boys, where the life expectancy is 30 years. Interestingly, the final output have been excellent. This is the reason why they have attempted to use this drug aganist Myotubular Myopathy condition which they have believed is the best quicker way to treat the genetic disease condition.

The scientists have administered drug to the sick mice with the same symptoms. An untreated sick mouse lived for 45 days on average whereas the treated mouse have even survived for over 400 days. The muscle strength was tripled and it has proved to recover 60% of the muscular deficit but it still not known whether the total absence of paralysis might be achieved if this molecule is given after birth as it is a genetic condition which starts during foetal development. This Drug became a boon for  those Who are suffering Myotubular Myopathy

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Sumathya Pulugurtha

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