Medications & Supplements (other than steroids)

The information on this page represents the recommended standard of care for Duchenne muscular dystrophy. Most of the care recommendations also apply to Becker muscular dystrophy, but at older ages. Most, but not all, people with Duchenne are males—but the care recommendations apply to both males and females with Duchenne.

If you don’t understand any of the medical terms and concepts, ask your healthcare providers. Take notes and ask questions during your clinical visits.

Medication & Supplement Facts to Remember

  1. Steroids are the only medicines known to improve strength in people with Duchenne.
  2. Other medications commonly prescribed for Duchenne include those to support the heart, breathing muscles, bone health, gastrointestinal symptoms, hormone levels, and to manage pain.
  3. You can help us gather more data by registering on The Duchenne Registry.

Safe & Unsafe Medications in Duchenne

PPMD’s Safe & Unsafe Medications Resource (download) lists medications that are frequently used in the management of Duchenne (see “Medications That May Be Prescribed in Duchenne”, below, for more information), as well as both over-the-counter and prescription medications used for other conditions not specific to Duchenne that you may encounter. Included in this resource are cautions that should be observed that are specific to patients living with Duchenne.

We encourage you to use this resource both as a reference, but also a means to track you/your child’s medications.

Download PPMD’s New Safe & Unsafe Medications Resource >

Use of Supplements in Duchenne

There are many different medicines and supplemental treatments that are used for Duchenne, although there is little agreement (among parents, researchers, and clinicians) about how useful they are.

The experts who created the Care Considerations guidelines reviewed published data on substances that are sometimes used for Duchenne treatment, to see if there was enough information to make recommendations.

To see a summary of all key studies looking at the use of supplements in Duchenne, please click here to download.

The experts found:

  • The use of oxandrolone, an anabolic steroid (a different type of steroid that is typically misused by athletes to become stronger), is not recommended.
  • The safety of Botox has not been studied for the treatment or prevention of contractures and is not recommended.
  • There was no evidence to support the wide use of creatine. A randomized controlled trial of creatine in Duchenne did not show a clear benefit. Anyone who is taking creatine and has kidney problems should stop taking it right away.
  • There is not enough information to make recommendations about other supplements or other drugs that are sometimes used in Duchenne treatment, including:
    • Co-enzyme Q10
    • Carnitine
    • Amino Acids (glutamine, arginine)
    • Anti-inflammatories/antioxidants (fish oil, vitamin E, green tea extract, pentoxifylline)
    • Herbal or Botanical Extracts

The experts agreed that more research is needed to make recommendations about supplements and other medications. They encouraged clinical trials on supplements and encouraged families to be involved in registries like The Duchenne Registry to gather more information.

Medications That May Be Prescribed in Duchenne

Below is a list of medications that may be prescribed by your primary care providers or neuromuscular teams. NO medication or supplement should be started without a discussion with your primary care provider or neuromuscular team.

Steroids (Corticosteroids)

Corticosteroroids (steroids) are medications that are commonly prescribed to people living with Duchenne somewhere around age 4 years. Steroid regimens will be managed closely by your Neuromuscular Specialist (NMS). For more information about considerations for taking steroids in Duchenne, please visit this page.


AMONDYS 45 is an antisense oligonucleotide indicated for the treatment of Duchenne in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 45 skipping. More information on types of mutations and exon skipping can be found here.


EXONDYS 51 is an antisense oligonucleotide indicated for the treatment of Duchenne in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 51 skipping. More information on types of mutations and exon skipping can be found here.


VILTEPSO is an antisense oligonucleotide indicated for the treatment of Duchenne in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 53 skipping. More information on types of mutations and exon skipping can be found here.


VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne in patients who have a confirmed mutation of the Duchenne gene that is amenable to exon 53 skipping. More information on types of mutations and exon skipping can be found here.

Cardiac Medications

Angiotensin-Converting Enzyme (ACE) Inhibitors (i.e. Lisinopril, Captopril, Enalapril)

These are typically the first type of medication prescribed by cardiologists in Duchenne. These medications work by helping the blood vessels coming from the heart to relax and open up, making it easier for the heart to pump blood out to the body. ACE inhibitors also lower the amount of water your body retains. This along with widened blood vessels decreases your overall blood pressure. ACE inhibitors usually have no side effects, especially if they are started at low doses and are increased slowly.

Angiotensin Receptor Blockers (i.e. Losartan)

Occasionally, patients will develop a cough from ACE inhibitors. If this happens, your cardiologist may choose to switch you to Angiotensin Receptor Blockers (ARBs). ARBs work similarly to ACE inhibitors, but do not cause cough as a side effect. ACE inhibitors are usually chosen over ARBS to start because they have been used a great deal in pediatric patients, and have been found to have very little risk. With both ACE-Inhibitors and ARBs, It is important to regularly check your blood pressure so it does not get too low.

Beta-Blockers (i.e. Propranolol, Metoprolol, Carvedilol)

These medications work by slowing the heart rate down. When the heart beats slower, it is able to fill and pump more effectively. Beta-blockers can make patients feel a little sleepy when they first start taking them. This sleepiness is usually less if beta-blockers are started at a low dose and increased slowly over time. It is helpful to note when increasing doses, to do it over the weekend – if the new doses is started on Friday, by Monday, any tiredness caused by the increased dose is usually gone. It is important to regularly check the heart rate, so that it does not get too slow.

Aldosterone Antagonists (i.e. Eplerenone and Spironolactone)

Eplerenone and spironolactone are in a class of medications called aldosterone antagonists. These medications are used to help lower blood pressure by decreasing the volume of blood in the body that the heart needs to pump and circulate. Early studies have also shown that eplerenone or spironolactone, when taken in combination with ACE inhibitors or ARBs, may delay the development of fibrosis in the heart muscle. It is hoped that delaying fibrosis, we will also delay the onset of heart dysfunction. In studies, treatment in younger patients seemed to lead to a longer delay in the development of fibrosis. As with ACE inhibitors, it is important to regularly check blood pressures, so that they do not become too low.

Gastrointestinal (GI) Medications

Stool Softeners (i.e. ducosate, Miralax, glycerin suppositories)

Constipation can be a very real, and quite debilitating problem for people living with Duchenne.  Not having a bowel movement at least every four days can lead to chronic issues with constipation. Chronic constipation may lead to other conditions including stomach pain, bloating, painful bowel movements, reluctance of the patient to want to move their bowels (because it hurts!), as well as painful tiny tears in the rectum and lower bowel (called “fissures”) that cause bleeding and pain. Chronic constipation can also lead to problems breathing when abdominal distention restricts your ability to take deep breaths.

Medications (such as the examples listed above) may be prescribed. Stool softeners do not do anything to increase the frequency of stool, but soften the consistency of the stool so that it is more easily passed. Concerns have been raised about the use of Miralax in children — consult your physician before using this common stool softener medication.

Proton Pump Inhibitors (i.e. Priolsec, Prevacid, Protonix)

People taking steroids often complain of gastroesophogeal reflux (also called reflux, acid reflux, or heartburn). Too much acid in the esophagus (which connects your throat to your stomach) can lead to the development of ulcers, which can cause pain and bleeding. Proton pump inhibitors prevent the production of acid in the stomach. Less acid in the stomach means less acid that may be available to go up into the esophagus and cause symptoms.

Antacids (i.e. Tums, Rolaids, Alka-Seltzer)

Antacids do not prevent the production of acid, but “neutralize” the acid that exists in the stomach. Neutralized acid does not cause burning. Therefore, if the neutralized acid does go up into the esophagus, there will not be a burning sensation.

Bone Health Medications


Bisphosphonates are medications given to treat osteoporosis (weakening, thinning bones).  Bisphosphonates can be given orally (by mouth) or through an IV. The goal of bisphosphonate therapy is to increase the density of the bones of the body, including the vertebrae in the spine. Increasing the density of bones puts you at less risk for a fracture.

Non-Bisphosphonate Medications

Forteo (Teriparatide)

Forteo/teriparatide, is a synthetic parathyroid hormone. The action of this medication is to regulate calcium metabolism to promote the growth of new bone. Forten is given by daily injection at home, usually for 24 months. Forteo/teriparatide is not approved for pediatric patients and includes a black box warning (strongly contraindicated) for children/young adults who have skeletons that are still growing/maturing (may cause the development of osteosarcoma, a malignant tumor on bone).

Prolia (Denosumab)

Denosumab belongs to a drug class called “monoclonal antibodies,” which act to prevent the breakdown and resorption of bone in the body. It is given by injection (at home) every six months. This medication is not yet approved in pediatrics.

Calcium Supplement

Calcium is important for keeping bones and teeth healthy.  There are recommendations for the amount of calcium you need daily, according to age. If you diet is found to be lacking in calcium, a supplement may be prescribed.

There are two forms of calcium supplements: calcium carbonate and calcium citrate. Both forms of calcium generally contain vitamin D as well, ranging from 200 – 400 IU/tablet.

Calcium Carbonate often comes in the form of antacid medication (Tums, etc.), and generally contain 200 – 400 mg of calcium/tablet. Using calcium carbonate may enable management of GI symptoms, as well as supplement calcium.

Calcium Citrate is better absorbed than calcium carbonate, especially in patients also taking H2 receptor agonist or a proton-pump inhibitor for gastric acid issues. Calcium citrate is slightly more expensive and comes in a rather large pill size, making it difficult for some patients to take.

Growth and Puberty Medications

Growth Hormone

The pituitary gland is a tiny gland in the brain that releases growth hormone. Long term steroid therapy may lead to short stature in people with Duchenne. There are some people who have low levels of growth hormone, known  as a “growth hormone deficiency.” Growth hormone deficiency is diagnosed by an endocrinologist who performs a blood test called a “growth hormone stimulation test.” If growth hormone deficiency is diagnosed, supplemental growth hormone may be prescribed. Growth hormone is given by injection (“shot”) over a period of time to help with growth. Discuss the risks and benefits of growth hormone before starting this therapy.

Some people who take steroids may be shorter than other people their age, but may not actually have a growth hormone deficiency. At this time, growth hormone is not recommended for people who do not have growth hormone deficiency, regardless of their height. Learn more about growth and puberty.


Testosterone is the “male sex hormone” that is responsible for the changes that happen to males during puberty (ie, increased size of the testicles, facial/axillary/pubic hair, etc.).  Testosterone also helps to strengthen bones. Long term steroid therapy may lead to delayed puberty in people with Duchenne. Not starting puberty when others your age start can be very emotionally difficult. People who do not start puberty by age 14 years should be referred to an endocrinologist for evaluation. If puberty is delayed, testosterone can be given by injection (shot), gels or patches.

Anesthesia and Pain Medications

There are many medications that may be used for anesthesia and pain.  All medications used for pain and medications should be used carefully. Because this area is quite complicated, we have created a page discussing all of these medications.


It is important for people with Duchenne to follow recommended vaccination guidelines. However, steroid therapy may affect the types of vaccinations you can receive. Please visit our vaccinations page to read more about which vaccinations are safe to receive while on steroid therapy.