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

There are different approaches to pharmacological management:

muscle

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

spine

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.

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