Practical DMD Course 07. Epigenetic Therapies in DMD

This course provides an up-to-date review of therapeutic strategies targeting epigenetic modulation in Duchenne muscular dystrophy (DMD), integrating the pathophysiological basis of the disease with recent advances in therapies aimed at modifying disease progression. The course reviews the main mechanisms involved in muscle degeneration associated with dystrophin deficiency, including chronic inflammation, fibrosis, impaired muscle regeneration and fibro-fatty replacement of muscle tissue. Within this context, the role of histone deacetylases (HDACs) as epigenetic regulators involved in disease progression and as potential therapeutic targets is examined. The course explores the biological rationale for HDAC inhibitors and the available clinical evidence regarding their impact on muscle regeneration, functional preservation and the progression of fatty muscle infiltration. It also reviews the main clinical studies conducted in patients with DMD, addressing functional outcomes, histological findings and imaging biomarkers. Finally, the course discusses aspects related to the safety, tolerability and clinical application of these emerging therapeutic strategies, as well as their potential future integration into combination treatment approaches and the multidisciplinary management of Duchenne muscular dystrophy.