Archived drug prevents Duchenne muscular dystrophy muscle loss in mice | DuchenneXchange

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Archived drug prevents Duchenne muscular dystrophy muscle loss in mice


A drug that showed promise in clinical trials for Duchenne muscular dystrophy (DMD) decades ago is back under the microscope and has now been shown to reduce muscle wasting in mice.

We looked at the literature and found a drug that was used back in the 1980s. But when the genetic cause of DMD was found, a lot of research and funding shifted toward diagnosis, so we continued from where this drug research was left, running trials in mice.

We’ve shown that the non-toxic drug adenylosuccinic acid (ASA) protects muscle attached to the skeleton against damage, and limits the replacement of this muscle with non-functional tissue, such as fat.

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