Leading the future of rare disease medicine for patients like Mason.

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Our goal is to provide medicines to those with limited options and to help patients face rare diseases head on, with courage and confidence.

COVID-19 Information

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.

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