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*:

muscle

Preserved muscle strength and motor function4,5

stairs

Delayed loss of ambulation1,4

lungs

Preserved pulmonary function1,4,5

spine

Avoidance of scoliosis surgery1,4,5

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:

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.

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