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