Combining EZH2 inhibitors boosts treatment effects for Duchenne muscular dystrophy

A Korean research team has proposed a new treatment strategy that could improve treatment effectiveness for Duchenne muscular dystrophy (DMD), a rare inherited disease for which steroid treatment is the standard of care.

The team further confirmed that the EZH2 gene is also overactive in patients with DMD, which is caused by abnormalities in the dystrophin gene. This confirms the possibility of maximizing the treatment effect while minimizing side effects by combining existing steroid treatment with EZH2 inhibitors.

Seoul National University Hospital said Friday that Professor Chae Jong-hee of the Department of Clinical Genomics at the hospital, Professor Choi Mu-rim of the Department of Medical Science at Seoul National University College of Medicine, and Jeon Eun-young, a Ph. D. course student, analyzed muscle tissue from patients and animal models of Duchenne muscular dystrophy and identified overactivation of the EZH2 gene as a key mechanism that triggers muscle fibrosis and inflammatory responses, suggesting the possibility of new therapies to inhibit it.

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KBR (https://www.koreabiomed.com))

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