At Santhera, we focus on providing treatment options for disorders that have a severe impact on the lives of the children and adults affected by them.
For more than a decade, we have been committed to advancing research in the field of mitochondrial diseases and are working to offer hope to the many thousands of patients for whom treatment options have been lacking.
Mitochondria are found within virtually every cell of the body and generate energy required by the cells to function.1–4
Mitochondrial diseases are often a result of inherited genetic mutations1,3 and typically affect organs with high energy requirements, such as the brain, muscles, eye, ear, heart, liver and the gastrointestinal tract.2–4 When mitochondria do not produce enough energy, cells within the organ do not function properly, become damaged and eventually die, resulting in the symptoms typically seen in these diseases.1–4
We are proud that, thanks to our development efforts, Raxone (idebenone) – the first pharmaceutical product for a mitochondrial disorder – was approved in the European Union in September 2015 for the treatment of patients with Leber’s hereditary optic neuropathy (LHON).
Our research on therapies in rare diseases is currently focused on developing treatment options for: