Myotonic Dystrophy Research

A group of scientists from the US have developed a mouse model which might enable them to find a treatment for people with myotonic dystrophy.

The scientists, from the University of Virginia, created mice carrying the DNA that causes myotonic dystrophy that could either be turned on or off. If the DNA was switched on the mice developed myotonic dystrophy, but if it was turned off the heart and skeletal muscle function of most of the mice fully recovered.

The researchers believe that their results provide evidence that the disease can be reversed by eliminating the toxic molecules (mRNA) that are produced when the faulty gene is present. These findings are an important step forward for developing a possible treatment.

The Muscular Dystrophy Campaign Director of Research, Dr. Marita Pohlschmidt, said,

“The results of the research presented by the team of Us scientists are encouraging for finding a treatment for myotonic dystrophy.

“The research shows that it might be possible to reverse the symptoms of myotonic dystrophy when the toxic molecule causing the condition is neutralised. The conclusions in this paper support our belief that in the future this devastating muscle disease will be treatable.

“The Muscular Dystrophy Campaign currently supports several research projects into myotonic dystrophy and we are therefore very interested to hear about this new development. However, although this research suggests a possible treatment technique for the future, there is still a lot that needs to be done before we can be sure that this will be successful.”

For further information about this research please contact our information line on 020 7720 8055.