Non-Steroidal Small Molecules in Duchenne Muscular Dystrophy: Focus on Givinostat

Non-steroidal small molecules offer a promising therapeutic approach in Duchenne muscular dystrophy (DMD) by targeting inflammation, fibrosis, and other disease-modifying pathways without the adverse effects commonly associated with long-term corticosteroid use. Givinostat, a histone deacetylase inhibitor, exemplifies this strategy by reducing muscle inflammation and preserving muscle tissue integrity. Clinical trials demonstrated that givinostat improved functional outcomes, slowed disease progression, and was generally well-tolerated, supporting its regulatory approval. However, limitations remain, including variable efficacy depending on disease stage, potential hematologic and hepatic side effects, and the need for long-term safety data. These therapies are typically considered as adjuncts or alternatives for patients who cannot tolerate steroids or require additional disease-modifying interventions. By expanding the therapeutic arsenal, non-steroidal small molecules like givinostat provide clinicians and families with more personalized options to optimize outcomes and quality of life for patients with DMD.