Duchenne Muscular Dystrophy (DMD) is the most common and devastating type of muscular dystrophy and is characterized by a complete loss of functional dystrophin, leading to progressive muscle weakness and wasting. DMD is a rare disease that affects mostly males born worldwide.

In boys and men with DMD, weakness of respiratory muscles leads to a progressive decline in their ability to move air into/out of their lungs, leading to sleep disturbances and respiratory infections, especially when patients have lost their ability to walk. Studies estimate 55-90% of patients with DMD die from pulmonary complications.(9, 10)

Acute respiratory failure can occur due to:

  • 1

    Compromised respiratory dysfunction complicated by mucus plugging and further weakening of inspiratory/expiratory muscles

  • 2

    Repeated pneumonias, hospitalizations and intubations

Decreased ability to cough leads to retained secretions and high risk of recurrent respiratory tract infections.

What is the purpose of this clinical trial?

  • 1

    Respiratory failure is one of the major causes of morbidity and early mortality in DMD, even with steroid use.

  • 2

    Progressive weakness of respiratory muscles leads to restrictive pulmonary disease.

  • 3

    Corticosteroids delay onset/progression of respiratory dysfunction, but do not prevent it (11).

  • 4

    Once patients begin to decline in pulmonary function, they decline in the same way irrespective of their steroid use status.

  • 5

    In a phase III study of DMD patients who were in respiratory decline and not taking steroids, idebenone was found to slow the loss of respiratory function. The SIDEROS study evaluates idebenone in DMD patients who are taking steroids(12, 13).