Once incurable muscle dystrophy gains approved drug cure

Research led by Professor Steve Wilton and Professor Sue Fletcher and licensed to Sarepta Therapeutics has obtained a new drug for Duchenne muscular dystrophy. The drug is rapidly gaining approval as time goes.

Primarily affecting males, Duchenne muscular dystrophy (DMD) is a hereditary disorder that results in muscle weakness and declining muscle mass. People who have this disease are usually bound to a wheelchair by the age of 12 and typically do not survive past the age of 20.

While the disease is fairly uncommon, DMD is the most frequently occurring of the children’s muscular dystrophies and was for some time believed to have no cure. Due to collaboration with Sarepta Therapeutics that go over the past 2 decades, were able to finally get the first approved therapy for DMD in 2016.

Another drug, golodirsen (Vyondys 53), has gained approval by U.S. Food and Drug Administration and although this indicates an evaluation of the current progress, the treatment will be accessible for patients in America.

Golodirsen was manufactured in the Perron Institute Laboratory and the license was given to Sarepta Therapeutics. The University of Western Australia. Prof. Wilton, Prof. Fletcher and their research team are now based at the Centre for Molecular Medicine and Innovative Therapeutics.

Both DMD treatments made by the Perth team take advantage if the cell machinery to manipulate cells into skipping the disease causing error, which would serve as a genetic white-out. Young males who received the treatment in the US has shown the ability to walk on their legs unaided in their late teens, while without the treatment they would have already required the aid of a wheelchair.  The inclusion of the treatment has therefore, made an astounding impact on the lives of those who were afflicted with the disease.