Our research

Our research on therapies in rare diseases is currently focused on developing treatment options for:

Neuromuscular Diseases

• Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscular degeneration and results in progressive muscle weakness, starting at an early age. Learn more

• Becker muscular dystrophy (BMD) is a progressive muscle wasting disease similar to Duchenne muscular dystrophy (DMD) but usually milder.
  

Pulmonary Diseases

• Cystic fibrosis (CF) is a rare, hereditary, life-threatening, progressive disease characterized by persistent lung infection and chronic inflammation thereby limiting the ability to breathe over time.

• Acute respiratory distress syndrome (ARDS) is a serious pulmonary condition in which fluid builds up inside the tiny air sacs of the lungs and surfactant breaks down. This prevents the lungs from properly filling with air and moving enough oxygen into the bloodstream and throughout the body.