When talking about DMD treatment, the set of medical and supportive actions used to slow disease progression, improve quality of life, and extend lifespan for people with Duchenne Muscular Dystrophy. Also known as Duchenne therapy, it brings together drugs, gene‑based approaches, and daily care routines. DMD treatment isn’t a single magic bullet; it’s a combination of options that work together.
One of the first entities you’ll hear linked to DMD treatment is Duchenne muscular dystrophy, the genetic disorder caused by mutations in the dystrophin gene. Because the root problem is missing or faulty dystrophin, modern therapy often focuses on gene therapy, which aims to deliver a functional copy of the gene or repair the mutation. Companies are testing viral vectors that carry mini‑dystrophin, and early trials show promising muscle strength gains. In parallel, exon skipping drugs like eteplirsen bind to specific RNA sequences, nudging the cell to skip over faulty exons and produce a truncated but workable protein.
Even with cutting‑edge genetics, corticosteroids remain a cornerstone of DMD treatment. Prednisone and deflazacort help preserve muscle strength and delay the need for braces or wheelchairs. The trade‑off is side‑effects such as weight gain and bone thinning, so doctors balance dose and timing carefully. Beyond drugs, physical therapy is essential; regular stretching, low‑impact exercise, and aquatic programs keep joints flexible and reduce contractures. Cardiac care also matters because the heart muscle weakens over time—routine echo monitoring and ACE inhibitors or beta‑blockers can stave off heart failure.
Respiratory support rounds out the treatment puzzle. As diaphragm muscles weaken, non‑invasive ventilation at night improves sleep quality and oxygen levels. Some families also use cough‑assist devices to clear secretions and cut down infection risk. Nutrition isn’t an afterthought either; high‑protein diets and calorie monitoring help maintain body mass when steroids increase appetite. When you combine these elements—gene therapy, exon skipping, steroids, physical therapy, cardiac drugs, and respiratory aids—you get a multi‑layered approach that addresses the disease from several angles.
All of this can feel overwhelming, but the good news is that the DMD community now has a wealth of resources. Below you’ll find articles that break down each therapy, compare medication options, and offer practical tips for daily living. Whether you’re a parent looking for the latest trial updates or a patient wanting to tune your exercise routine, the collection gives you actionable insight to make informed decisions about your DMD treatment journey.
Explore how Calcort (Deflazacort) measures up against common steroids, its benefits, side‑effects, cost and viable alternatives for conditions like DMD.