DM1 is the most common form of adult muscular dystrophy and there is currently no treatment for it. It is caused by a change in the DMPK gene that makes it much larger than it should be. To make proteins, genetic material needs to leave the cell’s nucleus ‒ where DNA is stored. However, when the DMPK gene is too large, it is unable to do this. This means it builds up in the nucleus, which isn’t good for the cell.
Recently, there have been suggestions that an antibiotic called erythromycin could help remove DMPK structures from the nucleus, meaning it could potentially be used as a treatment for DM1. A team of researchers led by Professor David Brook also found that another similar type of macrolide antibiotic called azithromycin (AZM), could work even better than erythromycin. However, more tests are needed to understand how this antibiotic works on DMPK structures and if it can be used to treat DM1.