We’re here to help patients, caregivers, and families
Become empowered with knowledge you can trust
Empowering and supporting patients, families and caregivers
- Understanding and Managing Behavior in Duchenne
- College Options for Individuals with Physical Disability
- Healthcare Transitions Nutrition Implications and Planning
A personal guide for navigating treatment
Engage, elevate, and empower people affected by rare disease
We provide resources and support to patients and their families throughout their journey with rare disease. 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.
North American Neurology Team
North American Gene Therapy Team
Clinical Patient Engagement Liaison
Global Patient Advocacy Team
Clinical Trials at PTC
PTC is conducting clinical trials in a variety of disease areas. New ones are continually being added.