Corticosteroid treatment in Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe degenerative disease that remains incurable. Its prognosis has been transformed by multidisciplinary care, which has significantly improved the life expectancy of patients. Corticosteroids have been used for over 20 years and have modified the care of young boys with DMD: They delay the loss of ambulation, support the gradual introduction of technical mobility aids, and reduce the frequency of scoliosis surgery. Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit-risk ratio favors treatment. If introduced early enough, when muscle degeneration is not too advanced, they prolong the ability to climb stairs, rise from the floor, walk, and bring the hands to the mouth. They have been suggested to contribute to delaying the onset of respiratory failure and cardiomyopathy. The well-known side effects of corticosteroids require specific management (to prevent obesity and osteoporosis) and/or adjustment of their dosage. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor.