FDA’s Review on Eteplirsen could Determine Quality of Life of Thousands
Food and Drug Administration controls the fate of so many people and the government agency can also deny children a treatment which might help them to walk.
Right now, the FDA advisory committee is reviewing an experimental drug called eteplirsen. It is a treatment for a strain of Duchenne muscular dystrophy and is designed by Sarepta based in Boston. The outcome of the review on Monday, could decide the quality of life for several thousands who suffer from the rare diseases.
Duchenne muscular dystrophy is a genetic disorder that weakens each body muscle. The condition that mainly affects boys, of around age twelve weakens their limbs and they can walk no longer. It also damages the heart and lungs over time. The disease is also fatal and most of those who suffer from this disease do not live more than twenty-five.
Eteplirsen fundamentally pumps out the protein thtat are missing in patients with Duchenne called dystrophin, as its skips over defective genetic code.
The trial for Sarepta began in 2011 and treated some boys of around nine years age, whose conditions with muscles and their abilities seemed to be deteriorating rapidly. They underwent treatment for four years and ten out of twelve children can still walk. Compared to that, in another group of eleven boys who were not treated, only one can walk still. There were no side effects or safety concerns reported.
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