On this five-week course from the European Joint Programme on Rare Diseases, you’ll explore the therapeutic development process from the unique perspective of rare diseases.

You will follow the journey from discovery to approved treatment, learn how therapeutic targets and drug candidates are identified and validated, and examine the special regulatory frameworks.

Describe the characteristics of translational research for rare diseases

Guided by experts in the field of rare diseases, you’ll examine current research projects, and learn the definitions and contexts, as well as hearing from patients directly involved in clinical research.

You will explore some of the specific challenges of translational research, gaining contextual insights into this vital area of medical research.

Learn how to move from bench to bedside

From first-in-man to post-marketing studies, you’ll trace each step of a clinical trial, learning to plan and design your trial and address any regulatory issues.

You’ll examine how to end a clinical trial and how to approach marketing approval and authorisation. You’ll also investigate data sharing and reuse post-trial.

By the end of this course, you’ll understand how, during the process of translational research, you build evidence that a treatment is safe and effective.

Available now!