ABSTRACT
Rare diseases (RDs) are defined as conditions affecting fewer than 200,000 patients for a given condition in the United States or less than 5 in 10,000 people in Europe. RDs are most often severely debilitating diseases associated with complex mixes of lifelong symptoms that lead to dramatically reduced life expectancies. Eighty per cent of orphan patients are children. At first glance, a patient affected with such a rare and severe disease should be perceived as unworthy of attention. When new therapies emerge in the preclinical field, only outstanding work and skills can allow for a successful translation from animal proofs of concepts and laboratory benches to patient's bedsides. LYSOGENE is now a clinical stage gene therapy company. It is developing a platform approach with a promising breakthrough to treat, and cure, life-threatening orphan diseases with a central nervous system component that currently have few or no treatment options.
