DMD treatments: what works now and what to ask your doctor

Here’s a fact that changes how families plan: treatments for Duchenne muscular dystrophy (DMD) have moved from purely supportive care to real disease-targeting options. That doesn’t mean there’s a one-size-fits-all cure — but knowing the current choices and the questions to ask can make a big difference.

First, get the exact genetic diagnosis. DMD treatments depend on the mutation type. Ask for a genetics report and bring it to a neuromuscular specialist. If you don’t have one, a specialist clinic or a Duchenne treatment center is worth seeking out.

Approved drug options you should know

Corticosteroids are the foundation for most kids with DMD. Prednisone and deflazacort (Emflaza) slow muscle decline and help prolong walking ability. They come with side effects—weight gain, bone thinning, mood swings—so plan bone density checks and talk about dose schedules that balance benefits and risks.

There are mutation-specific drugs called exon-skipping therapies. If your child’s mutation fits, drugs like eteplirsen (targets exon 51), golodirsen/viltolarsen (exon 53), or casimersen (exon 45) may be options. These aren’t for everyone, and their benefits vary, but they’re aimed at producing a working shorter dystrophin protein.

Gene therapy has advanced fast. Micro-dystrophin gene therapies (now available for some patients) deliver a smaller version of dystrophin via a viral vector. These can be game-changers for eligible kids, but they carry immune and safety considerations. Ask about long-term follow-up and eligibility screening.

Supportive care that really matters

Good care beyond drugs keeps people healthier longer. Regular physical therapy and stretching keep joints flexible and help delay contractures. Monitor breathing and heart function routinely—pulmonary tests and cardiac echocardiograms are standard. When breathing weakens, noninvasive ventilation and cough-assist devices reduce complications.

Bone health is a constant concern. Steroids increase fracture risk, so DEXA scans, vitamin D, and calcium should be on the checklist. Scoliosis can develop fast once walking stops—spine surgery timing matters, so discuss options early.

Clinical trials are active and varied. If approved options aren’t a fit, trials can offer access to new gene edits, systemic drugs, or combo therapies. Use registries like the Duchenne Registry and clinicaltrials.gov to find trials, and discuss trial eligibility with your specialist.

Practical checklist for families: 1) confirm genetic mutation; 2) see a neuromuscular center; 3) discuss steroids and bone care; 4) review mutation-specific or gene therapies; 5) set up regular lung and heart checks; 6) ask about trials and registries. Small steps taken early give the best chance to keep kids active and healthy longer.

If you want, I can help draft questions to bring to your next appointment or point you to resources and registries for DMD families.