Recent studies have also demonstrated the benefits of early glucocorticoid treatment, before significant physical decline.1-3
Preserved muscle strength and motor function4,5
Delayed loss of ambulation1,4
Preserved pulmonary function1,4,5
Avoidance of scoliosis surgery1,4,5
*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.
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
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 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.
(excluding UK and Ireland)
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