Patient & Caregiver Resources
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.
Insightful Moments™
Become empowered with knowledge you can trust
Get tips on participating in a clinical trial. Learn how drugs are developed. Explore different types of assistance that may be available to you. Welcome to Insightful Moments, an informative content series. Para obtener más información sobre Momentos de Claridad, por favor visite nuestro sitio web.

Navigating Duchenne™
Empowering and supporting patients, families and caregivers
Navigating Duchenne™ is an educational program series that addresses a variety of topics to help patients, families and caregivers navigate their journey with Duchenne muscular dystrophy. It empowers, supports, and creates community for patients, families, and caregivers. Our programs are available at no cost and consist of videos, in-person sessions and virtual webinars.
Navigating Duchenne topics have included:
- 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
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.

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.