Sarepta obtains positive results on DMD therapy, shares skyrocket 50%
Category: #health  By Saipriya Iyer  Date: 2018-06-21
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Sarepta obtains positive results on DMD therapy, shares skyrocket 50%

The shares of Sarepta Therapeutics Inc., a medical research & drug development firm, climbed up by 50% after the firm released initial results derived from clinical treatment on patients suffering from Duchenne muscular dystrophy (DMD), a rare degenerative genetic disease.

Reportedly, DMD causes muscle wasting in patients and can prove to be incurable after the patients attain the age of 30. The symptoms of ailment are observed in children between the age of three to five years with boys being more prone to the disease as compared to girls.

Sarepta Therapeutics had shared the experimental outcomes from three children, who were treated with the gene therapy during a phase 1/2a clinical test. The outcomes derived from the experiments on the first three patients registered in the phase 1/2a trial displayed an improvement in micro-dystrophin, a shorter kind of dystrophin gene. A mutation in this gene is witnessed in the patients affected from DMD, leading to the low production of protein dystrophin and eventually, muscle wasting.

Additionally, the phase 1/2a trials demonstrated that there was a substantial decline in serum creatine kinase enzyme in patients affected due to DMD after they underwent Sarepta’s gene treatment. For the record, creatine is an enzyme linked to muscle damage and is detected at an escalated level in patients suffering from DMD.

Dr. Jerry Mendell, the principal investigator of the phase 1/2a clinical study, has stated that the new gene therapy introduced by Sarepta Therapeutics drastically reduces creatine kinase levels and produces high proportion of protein dystrophin. He further commented that though the outcomes from the trials are preliminary, if they continue to deliver positive results through Sarepta’s gene therapy and are tested in more patients, they will prove to be a breakthrough in DMD treatment.



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Saipriya Iyer

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Saipriya Iyer

Saipriya Iyer develops content for Market Size Forecasters, Algosonline, and myriad other platforms. A computer engineer by profession, she ventured into the field of writing for the love of playing with words. Having had a previous experience of 3 years under her bel...

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