What will you learn?
By the end of the course, you will be able to...
✔ Explain how new therapeutic targets are identified
✔ Explain what is meant by hit identification and lead optimization
✔ Summarize the concept of drug repurposing and why it is particularly relevant to the field of rare diseases
✔ Explain how AI and data mining can accelerate drug discovery
✔ Discuss what "pre-clinical model systems" are and why they are used to study rare diseases and develop therapeutic approaches
✔ Summarize the concept of the "3Rs" and the ethical aspects of using animals to develop therapies
✔ Explain how clinical trials are conducted and sponsored
✔ Discuss the specific issues of clinical trials for rare diseases
✔ Describe the role of regulatory agencies and the regulatory framework for developing a treatment for rare diseases
✔ Discuss why patient involvement is key in each phase of therapeutic development, particularly in the field of rare diseases
✔ Explain the concepts of data use and sharing
Who is this course for?
This course is intended for researchers and students in medicine and health-related research fields, as well as health professionals interested in furthering their knowledge of translational research in rare diseases. It is also suitable for biotech and start-up developers and patient organization representatives who wish to deepen their understanding of current practices in therapeutic development for rare diseases.
This course was developed with funding from the EJP RD project. The European Joint Program on Rare Diseases is an initiative that has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement N825575.
