Certificate of Achievement
has completed the following course:
From Lab to Clinic: Translational Research for Rare Diseases
Foundation for Rare Diseases and European Joint Programme on Rare Diseases
This online course intends to give an overview of issues, challenges and opportunities in translating basic research findings into medical treatments and therapies, with a special focus on the development of treatments for rare diseases. It outlines how preclinical studies are performed in model systems in the laboratory, how these are translated into clinical trials and finally brought to the patients.
5 weeks, 4 hours per week
Magda Granata
European Joint Programme on Rare Diseases - WP16 Online Academic Course Coordinator
Foundation for Rare Diseases
Transcript
Learning outcomes
- Report how new therapeutic targets are identified
- Explain what is meant by hit identification and leads optimization
- Summarise the concept of drug repurposing and why it is particularly relevant in the field of rare diseases
- Describe what preclinical model systems are and why they are used to study rare diseases and to develop therapeutic approaches
- Summarise the concept of “3Rs” and the ethical aspects of using animals for therapy development
- Report how clinical trials are conducted and sponsored
- Debate the specific challenges of clinical trials for rare diseases
- Describe the role of regulatory agencies and the regulatory framework to develop a treatment for rare diseases
- Discuss why the implication of patients is key in each phase of the therapeutic development, especially in the field of rare diseases
- Explain the concepts of data use and data sharing
Syllabus
- The landscape of translational research
- The different phases and main stakeholders of translational research
- The specific issues encountered when developing a treatment for a rare disease and how they are overcome
- The different therapeutic approaches for the treatment of rare diseases
- The role and place of patients living with a rare diseases in this translational process
In association with
Issued on 28th April 2024
The person named on this certificate has completed the activities in the transcript above. For more information about Certificates of Achievement and the effort required to become eligible, visit futurelearn.com/proof-of-learning/certificate-of-achievement.
This certificate represents proof of learning. It is not a formal qualification, degree, or part of a degree.