We strive to revolutionize the lives of people living with rare diseases. We work in close partnership with caregivers, patient community, healthcare professionals, government authorities, and other stakeholders to build a brighter future for all. We relentlessly pursue sustainable development goals with passion, courage, teamwork, and innovation. We believe, from timely and accurate diagnoses to receiving effective treatments and finding supportive communities, no one should feel left behind.
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Our rare disease Mission
Our passion is Personal
As a family-owned and -run biopharmaceutical company, our goal is to make a real difference
in the life of every person and every family impacted by rare diseases.