Summary

• Duchenne muscular dystrophy (DMD) is highlighted as the most common and most rapidly progressive muscular dystrophy, with most patients losing the ability to walk by 12 years of age and requiring ventilatory support by 25 years of age.
• Before the use of cardioprotective drugs and respiratory muscle aids, respiratory complications were responsible for 80% of deaths and cardiac complications were responsible for approximately 20%. Survival past 30 years of age was rare but is no longer, with 10% to 40% of patients with DMD surviving to age 40 in centres that use non-invasive means of respiratory support.
• The development of acute respiratory failure, previously considered inevitable, can be preventable for most patients.
• Severe scoliosis and the need for corrective surgery can be avoided, and the need for mechanical ventilation delayed, by early and aggressive use of corticosteroids.
• With a few exceptions, all generalised muscle disease can be managed using the principles for managing DMD.