Patients
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.
