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Patients & Caregivers

We're Here to Help
We provide resources and support to patients and their families throughout their journey

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

A personal guide for navigating treatment

With our PTC Cares™ program, you can receive personalized support to gain access to treatment. Our case managers are here to help address any potential challenges.

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Insightful Moments™

Become empowered with knowledge you can trust

Educational support and resources for families living with rare disease. You will find programs and materials covering topics from navigating clinical trials to tips on how to handle stress.

  • Insightful Moments – a community education series that supports patients and families
  • Momentos de Claridad es una serie de programas y materiales educativos para la comunidad, creados para apoyar a los pacientes con enfermedades raras y sus familiares.
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Clinical Trials at PTC

PTC is conducting clinical trials in a variety of disease areas. New ones are continually being added.