Pharmacological treatments for the management of Duchenne muscular dystrophy (DMD)

There are different approaches to pharmacological management:


Glucocorticoids, a mainstay therapy, for the treatment of muscle strength and function1,2

Angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) for the treatment of heart disease3


Bisphosphonate therapy for the treatment of osteoporosis3

Hormone replacement therapy for the treatment of impaired growth, delayed puberty, and adrenal insufficiency1

Additional therapeutic strategies:*4

DNA helix

Mutation-specific therapies or gene replacement therapies aiming to restore dystrophin production

DNA helix

Muscle membrane stabilisation and/or upregulation of compensatory proteins which are structurally and functionally similar to dystrophin

DNA helix

Reduction of the inflammatory cascade and/or enhancement of muscle regeneration

*Some of these treatments have been approved by regulators while others are near, or in regulatory review or in clinical trials and might become available in the future1,4

Duchenne International Care Considerations highlight that physiotherapy and glucocorticoids remain the mainstays of treatment for Duchenne muscular dystrophy – click here to learn more.

1. Birnkrant DJ, et al. Lancet Neurol. 2018;17:251–267 [Part 1].
2. Bushby K, et al. Lancet Neurol. 2010;9:77–93.
3. Birnkrant DJ, et al. Lancet Neurol. 2018;17:347–361 [Part 2].
4. Mah JK. Neuropsychiatr Dis Treat. 2016;12:1795–1807.

Our Use of Cookies

In addition to cookies that are necessary to operate this website, we use optional cookies to provide website functionality and give you the best possible experience. We won't set optional cookies unless you enable them. For more detailed information see our Cookie Statement , which explains among others, how to set your cookie preferences and how to recall your consent.

Cookie Settings

Some cookies are essential, others help us improve your experience by providing insights into how the website is used. Select the cookie types listed below, and then save your preferences. Refer to our Cookie Statement for more information.

Strictly Necessary cookies
Strictly necessary cookies enable core functionality such as security, network management, and accessibility.

I accept the advertising/targeting cookies
Advertising/targeting cookies, which we use to track user activity and sessions so that we can deliver a more personalized service, and (in the case of advertising cookies) which are set by the third parties with whom we execute advertising campaigns and allow us to provide advertisements relevant to you.