Fulfilling our mission to help people live healthier, happier lives takes an unwavering devotion to scientific research and development. It takes a spark of innovation, kindled to a blazing fire of discovery. It takes empathy and compassion from patient-focused emissaries to carefully place scientific advancement into the hands of the people who need it the most: patients with high unmet medical need. And, in the field of rare disease science, it takes patience and tenacity.