What disease-modifying therapy slows disease progression in DMD and requires monitoring for side effects?

• A. Antioxidants (Vitamin E)
• B. Monoclonal antibody therapy
• C. Corticosteroids (prednisone/prednisolone or deflazacort)
• D. Cholinesterase inhibitors

Answer: (C)

Corticosteroids slow disease progression in Duchenne muscular dystrophy by dampening the inflammatory processes that drive muscle damage, helping preserve muscle strength and function for longer. They reduce muscle fiber breakdown, support motor abilities, and can delay loss of ambulation and decline in respiratory function, making them the best-supported disease-modifying option in DMD.

Because these medicines affect many body systems, they require ongoing monitoring for side effects. Growth in children may slow, affecting height velocity; weight gain and changes in fat distribution are common. Bone health can suffer, increasing the risk of osteoporosis and fractures. Glucose tolerance can worsen, potentially leading to diabetes. Blood pressure may rise, mood and behavior can change, and cataracts can develop with long-term use. Regular follow-up focuses on tracking height and weight, growth velocity, bone health, glucose regulation, blood pressure, mood and behavior, and eye exams, with dosing adjustments to maximize benefit and minimize harm.

Other options do not have this proven disease-modifying effect in DMD; they are not standard therapies for slowing disease progression in this condition.