We use our experience, insight, and commitment to move the rare disease community forward. Our goal is to provide medicines to those with limited options and to help patients face rare diseases head on, with courage and confidence.
We are committed to improving the lives of people with rare and ultra-rare genetic diseases through new medicines, educational initiatives, patient advocacy, research, and access to information.
Our diverse portfolio of approved medicines and investigational therapies is advancing first- or best-in-class treatments for diseases that exhibit a clear biology for treatment and are severely underserved by current standard of care.
As the COVID-19 pandemic continues to evolve, we are focused on the health and safety of our community including patients, caregivers, healthcare providers, partners, and employees. For patients specifically, this means that we are committed to providing an uninterrupted supply of our medicines to the patients who rely on them.