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 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 boys have lost their ability to walk. Studies estimate 55-90% of boys with DMD die from pulmonary complications.

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.

Why do we want to bring idebenone to the market?

  • 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.

  • 4

    Risks and side effects of steroids limit the possibility of long-term use.

  • 5

    Not all boys respond to steroids in the same way.

  • 6

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

  • 7

    Idebenone is found to slow the loss of respiratory function in patients with DMD.