Duchenne muscular dystrophy: Causes, treatments, and what you need to know

When a child starts falling more often, struggling to climb stairs, or walking on their toes, it might not just be clumsiness. It could be Duchenne muscular dystrophy, a genetic disorder that causes progressive muscle degeneration and weakness, primarily in boys. Also known as DMD, it’s the most common and severe form of muscular dystrophy in children, usually showing up between ages 2 and 5. This isn’t just about weak muscles—it’s about the body’s inability to make dystrophin, a protein that keeps muscle cells intact. Without it, muscles break down faster than they can repair, leading to loss of walking ability by the early teens and serious heart and breathing complications later on.

While there’s no cure yet, treatments have improved dramatically. Corticosteroids, like prednisone and deflazacort, are the standard of care. These drugs don’t fix the missing protein, but they slow muscle loss, help kids stay walking longer, and reduce the risk of spinal curvature. Another key player is gene therapy, a newer option that delivers a functional version of the dystrophin gene. It’s not for everyone—cost, age, and immune response matter—but for some families, it’s changed the trajectory of the disease. Physical therapy, breathing support, and heart monitoring are just as critical as any pill or injection. Many parents also look into supplements like coenzyme Q10 or creatine, but the science is still thin. What works for one child might not work for another, and timing matters—starting treatment early makes a big difference.

What you’ll find in the posts below isn’t just a list of drugs. It’s real-world insight into how medications like corticosteroids are used, what side effects to watch for, and how treatments for other muscle and nerve conditions—like tardive dyskinesia or muscle spasms—sometimes overlap in approach. You’ll see how people manage chronic conditions with daily meds, what emergency signs to never ignore, and how to navigate treatment choices when options are limited or expensive. This isn’t theoretical. It’s what families are dealing with every day, and the information here is meant to help you ask better questions, spot red flags, and understand what’s really happening behind the prescriptions.