Rare University

The aim of this course is to help you understand the drug development process; where the journey begins and the key stages of the process, who is involved along the way and how they contribute to the process, and what challenges must be overcome. As you learn about the drug development process in this course,…

This course has been developed specifically for people who need to learn more about a genetic disease that affects them or someone they love. Whether you are at the very start of your journey or further along, this course is designed to help you in three ways; to help you understand more about the concepts…

This is a hopeful time for the rare disease community. Our understanding of the causes of rare diseases is rapidly growing, along with the development of innovative approaches to treatment. The ability to collect and share data is at the heart of these advances. That’s why patients and advocacy groups are taking on the task…

Whether you are creating a contact registry, a clinical patient registry, a natural history study, or a biobank, one of your first steps will be to create a process that allows you to collect, store, and share data in a way that protects the rights and privacy of each participant. The purpose of this module…

Developing treatment for rare diseases is always a team effort. As an advocacy leader, you have an essential role to play in convening this team. In this module, you’ll meet Dr. David Fajgenbaum and learn about the 8-Step Collaborative Research Network he created to advance treatment for Castleman Disease. Next, you’ll draft an action plan…

A strong data program is one of the most valuable assets a rare disease organization can possess. It provides a solid foundation for strategy development and decision making. It makes your organization attractive to funders, investors, and research partners. It can even reveal new solutions to seemingly unsolvable problems. This module assembles stories of advocacy…