Treatments for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a genetic disorder in which the muscles in the body weaken and break down over time. This is due to a mutation, or change, in the DMD gene. The DMD gene is responsible for producing dystrophin, a protein that keeps the muscles intact. Without dystrophin, muscles weaken and deteriorate.¹

DMD is a rare disease that mostly affects people assigned male at birth. People with DMD begin to show symptoms in early childhood, typically before age 6.¹

How is Duchenne muscular dystrophy treated?

There is currently no cure for DMD. But several treatment options can help manage the symptoms, improve quality of life, and extend life expectancy.^1-3

Treatment options for Duchenne muscular dystrophy include:^1-3

• Steroids
• Nonsteroidal drugs
• Gene therapy
• Physical therapy
• Cardiac care
• Respiratory care

Steroids

One of the main treatments for DMD is steroid drugs (corticosteroids). Steroids are commonly prescribed to people with DMD to help slow the progression of muscle weakness. They help reduce the risk of scoliosis (curving of the spine) and delay heart problems. These are common complications of DMD. Steroids also can help people maintain lung function and overall strength.^1,2

Examples of steroids commonly prescribed for DMD include:^1,2

• Prednisone
• Deflazacort (Emflaza®)
• Vamorolone (Agamree®)

Like all drugs, steroids can come with a number of side effects, such as:^1,2

• Weight gain
• Bone thinning
• Mood changes

Nonsteroidal drugs

Researchers are exploring nonsteroidal drugs for DMD treatment because they may cause fewer side effects than steroids. Nonsteroidal drugs reduce inflammation and loss of muscle.³

In 2024, the US Food and Drug Administration (FDA) approved the following nonsteroidal drug to treat DMD:^1,3

• Givinostat (Duvyzat™)

Side effects of givinostat can include:^1,4

• Diarrhea
• Stomach pain
• Nausea/vomiting

Gene therapy

Recent advancements have been made in gene therapy for DMD. Gene therapy aims to address the root cause of the disease by targeting the mutated gene responsible for DMD. It delivers a shortened version of the dystrophin gene, which is missing in people with DMD.^1,3

The FDA has approved several gene therapy drugs, including:^1,3

• Delandistrogene moxeparvovec-rokl (Elevidys®)
• Viltolarsen (Viltepso®)
• Eteplirsen (Exondys 51™)
• Golodirsen (Vyondys 53™)
• Casimersen (Amondys 45)

Gene therapy offers hope by potentially slowing the progression of the disease and improving muscle function. However, it is not a cure for DMD.^1,3

At this time, gene therapy drugs are available only to people with certain genetic profiles. And the immune system’s response to these drugs can make them less effective. For some people, this therapy can be used only once. More research is needed to learn how to make gene therapies more accessible and effective.^1,3

Physical therapy

Physical therapy plays a crucial role in managing DMD. It helps maintain muscle strength and flexibility.²

Physical therapists design exercise programs that include stretching and low-impact exercises like cycling and swimming. This helps keep the muscles as healthy as possible.²

Assistive devices such as leg braces, standing frames, and wheelchairs might also be recommended. These can help people move more easily on their own.²

Cardiac care

DMD often affects the heart muscle. This commonly leads to cardiomyopathy, a disease of the heart muscle. People with DMD need regular heart monitoring.²

Doctors may prescribe medicines that manage heart function and reduce the risk of cardiomyopathy, such as:²

• Angiotensin-converting enzyme (ACE) inhibitors
• Beta-blockers
• Digoxin

Respiratory care

As DMD progresses, respiratory muscles weaken. This makes breathing more difficult. Respiratory care can help and might include:²

• Using an oxygen mask to help with breathing
• A tracheostomy, in which a surgeon makes a hole in the neck and into the windpipe to provide an airway
• Mechanical ventilation in more severe cases

Other things to know

Managing DMD requires a multidisciplinary approach. A person with DMD will most likely have many healthcare specialists working together to create a treatment plan. Their team may include:²

• Neurologists
• Cardiologists
• Pulmonologists
• Physical therapists
• Genetic counselors
• Dietitians or nutritionists

Regular follow-ups and monitoring are vital for people with DMD. Medicines and treatment plans may need to be adjusted as the disease progresses.²

These are not all the possible side effects of steroids, nonsteroidal drugs, and gene therapy drugs. Talk to your doctor about what to expect when taking these drugs. You also should call your doctor if you have any changes that concern you when taking these drugs.