A few drugs exploiting exon‐skipping technology achieved the Food and Drug Administration (FDA) approval, but their therapeutic potential is limited to patients with specific mutations in the dystrophin gene.[18] In 2023, the FDA approved the first gene therapy‐based drug under the name of Elevidys.[19] Nonetheless, even if gene‐therapy approaches increase the life expectancy of DMD patients, combinatorial therapy with pharmacological agents is still necessary to delay DMD progression, manage DMD symptoms, and ameliorate patients’ quality of life. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.