Pharmacological therapy: Learn more about treatment with glucocorticoids

Along with physiotherapy, treatment with glucocorticoids remains a mainstay of Duchenne muscular dystrophy treatment and it is recommended that individuals should continue taking glucocorticoids after loss of ambulation.¹

Recent studies have also demonstrated the benefits of early glucocorticoid treatment, before significant physical decline.^1-3

The long-term effects of glucocorticoid therapy have been shown to include*:

Preserved muscle strength and motor function^4,5

Delayed loss of ambulation^1,4

Preserved pulmonary function^1,4,5

Avoidance of scoliosis surgery^1,4,5

Improved survival⁴

*compared to untreated patients

The 2018 International Care Guidelines recommends following an initial consultation with the family, a discussion regarding side effects and a nutritional consultation should occur before any glucocorticoid treatment is initiated.¹ Well documented side effects of long-term corticosteroid use can include; weight gain and obesity, acne and warts, cushingoid features, growth retardation and delayed puberty, cataracts, immune/adrenal suppression, glucose intolerance, hypertension, adverse behavioural changes, gastro-oesophageal reflux, peptic ulcer, gastritis, osteoporosis and myoglobinuria.⁶

Click here for the International Duchenne Care Considerations summaries.

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 function^1,2

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

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

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

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 future^1,4

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