No cure currently exists for Duchenne muscular dystrophy. However, treatments can help improve muscle function.
The most promising type of treatment is a new gene therapy called Elevidys (delandistrogene moxeparvovec), which the FDA approved in June 2023. Many children between the ages of 4 and 5 who are diagnosed with DMD are eligible for this treatment. It is given as a single intravenous dose. This therapy delivers a gene that will cause muscles to produce a shortened version of the dystrophin protein.
While long-term benefits of Elevidys are not yet known, clinical trials suggest it might improve muscle function. Experts hope Elevidys will also extend the life expectancy of patients with DMD. UK HealthCare is proud to offer this groundbreaking treatment, and our multidisciplinary team of experts works together to ensure that it is delivered safely.
Elevidys treatment can lead to injury of the liver, so patients will have liver function monitored weekly for the first three months. Inflammation of the heart muscle can also occur, and weekly blood tests will be given for the first month to check levels of a heart protein called troponin-I.
The most frequently used treatment to date for DMD is steroid treatment. The steroids used for DMD are corticosteroids. They help maintain muscle strength, slow the progression of muscle damage and prolong the period of time that children can walk.
Typically, doctors discuss steroids during the early stages of diagnosis. Most kids begin steroid use before they experience significant weakness, which is usually around age 4 or 5.
- Prednisone: This is the most commonly used medication for the treatment of DMD. Known for its anti-inflammatory properties, prednisone helps delay muscle weakness progression. It comes in tablet and liquid form and is taken orally on a daily basis.
- Emflaza (deflazacort): This corticosteroid, which is available as a tablet or liquid and taken by mouth, is for kids ages 2 and older. Deflazacort is a daily medication that helps delay DMD progression by helping children maintain muscle function and strength. In addition to preserving muscle function in the arms and legs, studies have shown the deflazacort helped preserve lung function and delay scoliosis.
- Agamree (vamorolone): This liquid medication is taken by mouth once daily by children ages 2 and older. Vamorolone is a corticosteroid that helps improve muscle function and strength and reduce inflammation. This drug has promising results, as a study revealed that boys with DMD who took a specific dose of this medication were able to stand faster from a lying position, walk a greater distance and run faster than those who received a placebo.
To use an analogy, DNA is basically a giant instruction book for building all the parts of your body. Exons are an important chapter that explains how to make specific parts of a protein.
To function properly, exons must connect in a specific sequence, like a puzzle. When portions of the exons are missing, the rest of the exons can’t connect. When this happens with the dystrophin protein, it causes DMD.
During exon-skipping therapy, a medication given through a vein creates “patches” to skip over the damaged exons. This allows the body to produce more dystrophin protein.
While exon-skipping therapy doesn’t cure DMD, it may help. In most cases, this therapy is given weekly or every other week at our pediatric infusion center. The infusion can take up to an hour.
