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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.
Insightful Moments

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
To learn more about Navigating Duchenne, please contact Malisa Rust, patient engagement liaison at mrust@ptcbio.com
Navigating Duchenne

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.
PTC Cares logo

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.