Thursday, January 12, 2012

One Step Closer to the End of Duchenne

You may have seen them on UK's X-Factor. The Lloyd family told their story prior to the contestants singing for their charity event. Twins, Dan and Sam, and even their younger brother, Tom, have all been diagnosed with DMD, or Duchenne Muscular Dystrophy. Duchenne is but one type of Muscular Dystrophy that affects about 1 in 3,000 males in the world, and about 100 per year in the U.K.

The way that it debilitates is by a lack of a protein called Dystrophin. This leads to a breakdown and loss of muscle cells. Professor George Dickson of the School of Biological Sciences at Royal Holloway and researchers have found that gene based drug treatment, drug AVI-4658, has been effective in restoring Dystrophin. Of course only 7 of 19 patients studied actually recovered the dystrophin, that 37% is huge considering there was no course of treatment prior and this is a first step towards an actual 100% cure.

Hear about it:

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