Rare diseases

At Santhera, we are passionate about providing treatment options for patients with rare diseases, specifically in the area of neuromuscular and pulmonary conditions – diseases that have a severe impact on the lives of affected individuals.

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
  • Congenital muscular dystrophy (CMD) is a group of inherited neuromuscular condisions that causes progressive and potentially life-threatening muscle weakness, affecting frequently newborns and children. Learn more

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.