DMD Gene Therapies and Treatments: How Do They Work?

Medically reviewed by Madison Saxton, PharmD
Written by Maureen McNulty
Posted on May 18, 2023

  • Multiple treatments can help relieve the symptoms of Duchenne muscular dystrophy (DMD), although none can cure or prevent the condition.
  • DMD treatments often include a mix of medications, surgery, physical therapy, respiratory treatments, mental health treatments, and exon-skipping and gene therapies.
  • Researchers are examining new DMD therapies in research studies.

Although Duchenne muscular dystrophy can’t be cured, many treatment options can help protect the health of your child or loved one as they live with this genetic disease. In particular, exon-skipping and gene therapies are promising new types of treatment for people with certain genetic mutations.

DMD is a rare genetic disorder that causes muscle weakness and degeneration over time. It’s caused by a mutation (change) in the dystrophin gene, which provides instructions for producing a protein called dystrophin. This protein acts like an anchor that holds muscle cells together, keeping them strong and protecting them from injury.

Current DMD Treatments

DMD treatment plans often aim to slow disease progression, minimize symptoms, and treat complications — additional medical conditions that occur alongside DMD. Overall, DMD treatments help people with this neuromuscular disease experience a higher quality of life.


Corticosteroids are an important part of treating DMD. These medications, including prednisolone (Omnipred) and deflazacort (Emflaza), can help prevent DMD from worsening too quickly. People who use these medications may maintain muscle strength, heart health, and breathing abilities for longer periods of time. They may even live longer.

Notably, corticosteroids can cause side effects like hormone imbalances, which can slow growth or delay puberty. Hormone therapy may help correct these issues.

Many people with DMD have cardiomyopathy (disease of the heart muscle), so doctors recommend medications that can help keep the heart working as it should. Drugs like ACE inhibitors, beta-blockers, and angiotensin-receptor blocker (ARBs) help slow down cardiac muscle damage for people with DMD. Health experts often recommend that children with DMD start taking heart medications by the time they turn 10.

When DMD weakens the bones, doctors may recommend bisphosphonates, such as alendronate (Binosto and Fosamax), zoledronic (Reclast and Zometa), and pamidronate (Aredia). These medications strengthen the bones and reduce the risk of fractures.

Adults with DMD may be able to boost bone health using medications like teriparatide (Forteo) and denosumab (Xgeva).


Surgery is sometimes a necessary part of DMD treatment. People with heart complications may need a surgical procedure to implant a pacemaker device. If bone problems such as a curved spine lead to breathing problems, surgery may be able to correct these issues.

Surgical procedures can also help treat contractures — a shortening and hardening of the muscles and tendons.

Exercise and Physical and Occupational Therapy

Getting physical activity is an important part of staying healthy while living with DMD. It helps boost flexibility, maintain muscle strength, and improve other measures of health. Exercise can help prevent contractures from developing and can slow down muscle atrophy (shrinkage of the muscle fibers). Make sure to talk to your health care team before trying any new exercises.

Most people with DMD go to physical therapy. This treatment may help children with the condition move around more easily and build strength. Physical therapists can demonstrate stretches or provide workout guidance to help treat some of the most common DMD issues.

Occupational therapy can also help those with DMD deal with everyday tasks. For example, if DMD symptoms prevent you from doing the things you want to do at home, school, or work, an occupational therapist may be able to help you figure out other ways of accomplishing those tasks.

Respiratory Treatments

DMD often leads to respiratory problems (trouble breathing in enough oxygen). These issues can be treated with several therapies from a pulmonologist or respiratory therapist.

Muscle weakness in DMD may lead to an inability to cough out mucus or foreign substances. However, coughing can be made easier with the help of manual assisted coughing (a technique in which another person helps squeeze your chest or abdomen while you cough) or a cough assist device (a machine that helps move air into and then quickly out of your lungs, mimicking a cough).

Noninvasive ventilation can also help get more air into the lungs. During this treatment, air is delivered into your body through a mask or nasal tube. If DMD worsens, doctors may recommend invasive ventilation, in which a tube is surgically placed into the windpipe to assist with breathing.

Mobility Aids

Walking is a challenge for most people with DMD by the time they reach their teenage years. However, it’s important to get some activity, as this will help boost muscle and bone health.

Various types of mobility aids can help people with DMD walk and move around more easily. Some effective options include:

  • Braces
  • Walkers
  • Standing frames
  • Shower chairs
  • Mechanical lifts
  • Wheelchairs

Many children with DMD begin using a wheelchair gradually over time. For example, you may initially only use a wheelchair when you have to travel longer distances, and eventually transition to using a wheelchair full-time. Wheelchairs often help people with DMD experience more independence and may help boost energy levels.

Treatments That Target Mental and Social Health

Many children with DMD have poor cognitive or social skills. They are also more likely to have other conditions that affect development, such as attention-deficit/hyperactivity disorder (ADHD). DMD medications and the stress of dealing with an ongoing health problem can also affect mental health.

Therapy and counseling may help people with DMD improve their mental health, learn new social skills, and experience fewer symptoms of psychiatric conditions. Individual therapy, as well as therapy that involves the whole family, may be useful. Therapists or social workers may also be able to help make sure that the needs of a child with DMD are met at school or within the workplace.

Exon-Skipping Drugs

Some newer medications target the gene mutations that cause DMD. DMD develops due to mutations in what’s called the dystrophin gene — sometimes called the DMD gene. When this gene contains abnormalities, it can’t send proper instructions to muscle cells to make enough of the dystrophin protein. Because this protein is necessary for keeping muscle cells working properly, too little dystrophin leads to muscle damage.

People with certain gene mutations may be able to try exon-skipping drugs. These gene-editing treatments are useful when people have a DMD gene mutation that leads to an abnormality within a certain exon (segment of a gene). As a muscle cell “reads” the abnormal dystrophin gene, exon-skipping drugs force the cells to ignore the mutated part, leading to increased levels of normal dystrophin protein within cells.

The first exon-skipping drug for DMD, Exondys 51 (a formulation of eteplirsen), was approved by the U.S. Food and Drug Administration (FDA) in 2016. This medication can be used by the 1 out of 7 people with DMD who have mutations in exon 51 of the dystrophin gene. It won’t help people with mutations in other parts of the gene.

In 2019, the FDA approved another drug called Vyondys 53, a formulation of golodirsen. This drug may help those with mutations in exon 53 of dystrophin. The agency approved another medication, Viltepso — a formulation of viltolarsen — in 2020 to treat mutations in the same part of the gene. These medications could be useful for up to 8 percent of people with DMD.

Another 9 percent of people with DMD have mutations in or near exon 45 of the dystrophin gene. These individuals may be able to use the exon-skipping drug Amondys 45, a formulation of casimersen, which was approved in 2021.

Gene Therapy and New DMD Treatments

Researchers are continually studying DMD and developing new treatments that can help people with the condition live longer.

Although the FDA has approved several exon-skipping drugs for DMD, each medication works only for people with a mutation in a specific location. Scientists are continuing to develop additional exon-skipping drugs that target DMD mutations in other places within the gene.

Researchers and scientists are also exploring therapeutics that work in new ways to treat DMD. For example, multiple groups are trying to develop gene therapy treatments that can insert a healthy version of the dystrophin gene into muscle cells, helping these cells produce normal dystrophin protein.

Researchers aren’t only studying potential gene therapies for DMD. They are also working on new treatments that may:

  • Regrow muscle tissue
  • Use stem cells to repair damaged muscle and improve muscle function
  • Turn on other genes that help prevent muscle injuries
  • Prevent and treat muscle inflammation
  • Stop scar tissue from building up within muscles
  • Boost heart health

You may be able to access new treatment options by participating in a clinical trial. Organizations like the Muscular Dystrophy Association maintain lists of relevant clinical trials to make them easier to find.

Talk With Others Who Understand

On myDMDcenter, people with Duchenne muscular dystrophy and their loved ones come together to gain a new understanding of DMD and share their stories with others who understand life with DMD.

Do you or your loved one with DMD take medication for your symptoms? What has your experience been like? Share more in the comments below.

Posted on May 18, 2023
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Have You Or Your Loved One With DMD Tried Any Symptom Medications? Share Your Experiences.
July 27, 2023 by myDMDcenter
Madison Saxton, PharmD obtained her Doctor of Pharmacy from Lake Erie College of Osteopathic Medicine (LECOM) in Bradenton, Florida. Learn more about her here.
Maureen McNulty studied molecular genetics and English at Ohio State University. Learn more about her here.

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