Based on its efficacy in a mouse model of Steinert’s disease (DM1), a Japanese team evaluated erythromycin (MYD-0124) against placebo in 30 DM1 patients treated for six months.
- The treatment was well tolerated.
- Two out of eleven biomarkers (splicing abnormalities of the MBNL1 and CACNA1 genes) were improved in the treated group compared with the placebo group.
These results may be used to develop the protocol for a new Phase III trial to determine the efficacy or otherwise of erythromycin in DM1.
