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We’re here to help patients, caregivers, and families

Our commitment to people and their loved ones living with rare disease runs deep. Providing reliable information and education on rare disease is just one more way we strive to support your journey.

Engage, elevate, and empower people affected by rare disease

We work with advocacy groups around the world to help raise awareness of the unique challenges our community faces. Through collaboration, compassion, and partnership, we provide support that empowers.

Patient associations can connect with our patient advocacy team on email PatientAdvocacy@ptcbio.com.