Health Care Professionals


Santhera is passionate about developing new treatments for patients with rare diseases. Our focus is on the development of treatments for neuromuscular and pulmonary diseases that currently lack treatment options.

Our DMD pipeline

Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa® (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to exclusively in-license vamorolone, a first-in-class anti-inflammatory drug candidate with novel mode of action, in all indications and all countries worldwide except Japan and South Korea. Vamorolone is currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids.