DMD treatment: what works now and what to ask about

If someone in your life has Duchenne muscular dystrophy (DMD), you want clear steps, not confusion. Treatment has changed a lot: there are standard medicines that slow decline, mutation-specific drugs and gene therapies for some patients, plus routine care that makes daily life better. Below are practical, no-nonsense points you can use at a clinic visit or when researching options.

Medical treatments you should know

Corticosteroids (prednisone or deflazacort) remain the first-line therapy. They can slow muscle loss, help keep walking longer, and reduce some complications — but they come with side effects like weight gain and bone thinning. Ask your doctor about dose strategies and monitoring (weight, blood pressure, growth and bone health).

Mutation-specific therapies exist for certain genetic changes. Exon-skipping drugs (for example, eteplirsen) target specific DMD gene mutations and may help some patients. Gene therapy advances are real: some micro-dystrophin gene therapies have reached approval or late-stage trials. Whether your child is eligible depends on the exact genetic test result, so getting a confirmed molecular diagnosis is essential.

Other supportive drugs focus on heart and lung health. Early cardiology care with ACE inhibitors or beta-blockers helps protect the heart. Pulmonary teams monitor breathing and may recommend cough-assist devices or noninvasive ventilation as needed.

Supportive care, daily living, and trials

Physical therapy and occupational therapy are practical: regular stretching, low-impact exercise, night splints, and adaptive equipment slow contractures and keep function longer. Talk to PT about a realistic home program — short, consistent sessions beat occasional marathon workouts.

Surgery can help with scoliosis and loss of ambulation; timing matters. Respiratory and cardiac clinics should be part of routine care from an early age. Vaccinations (flu, COVID, pneumococcal) and bone-health checks (DEXA scans) are simple things that reduce complications.

Want trials? Check clinicaltrials.gov and ask neuromuscular centers about open studies. Patient registries and advocacy groups (for example, Parent Project Muscular Dystrophy) often list trials and resources. Before joining a trial, ask about risks, long-term follow-up, and how the study will affect daily life.

Practical next steps: 1) confirm the exact DMD gene mutation if you haven’t, 2) get care at or connected to a neuromuscular center, 3) start or review a steroid plan with clear monitoring, 4) set up regular heart and lung checks, and 5) ask about trials if you want to consider mutation-specific or gene therapies. Keep notes from each visit and bring questions — that makes appointments far more useful.

If you want, I can help draft a short question list to take to your next clinic visit or summarize current trials for a specific mutation. Which would be more helpful right now?