PTC Mission

We know that when it comes to rare disorders, every day matters, and every moment counts. It’s why we are committed to making progress in rare disease through clinical trial research, each and every moment of every day.

Find a PTC Trial

What Are Clinical Trials?

A clinical trial is a research study used to validate a new treatment or therapy in volunteers young and old. Through clinical trials, researchers and healthcare professionals are able to determine if a treatment is safe and efficacious, and how it can be used to treat a specific disease or condition.

Watch the video above to learn more about what is involved in a clinical trial, and why they are so important.

Why Participate in a Clinical Trial?

By participating in a clinical trial, you are taking an active role in the development and approval of new medicines and treatments. People who volunteer in clinical trials provide the medical community with valuable research and data that helps to facilitate the development of new and potentially life-saving therapies.

Watch the video above for more information about why to participate in a clinical trial.