Alexion is focused on providing life-transforming treatments to patients with severe and life-threatening rare diseases for which there are few, if any, effective treatment options.
Many rare and ultra-rare diseases are severe, chronic and progressive, with high mortality rates and a profound impact on patients, families and society. Severe and ultra-rare diseases often present unique public health challenges. Typically, few researchers or companies explore such diseases. It is also highly likely that very few physicians are familiar with diagnosing and treating these conditions, leading to missed, delayed or inaccurate diagnosis and thereby delaying treatment, even when an approved, effective therapy is available.
Alexion has a proven track record in overcoming these hurdles on behalf of patients, and has significant experience in developing and delivering breakthrough therapies for patients with severe and life-threatening ultra-rare diseases.