Innovative Therapies and Personalised Medicine for Rare Diseases

Explore innovative personalized therapeutic approaches for rare diseases

In this five-week course of the European Joint Programme of Rare Diseases, you will have the opportunity to explore the most recent therapeutic treatments developed within medical research, such as: gene therapy, protein-based treatments, regenerative medicine, cell therapy and tissue engineering.

Guided by experts in the field, you will gain insight into how these approaches are developed and their potential applications, especially in the field of rare diseases, where they are providing unprecedented opportunities to treat, modify, reverse, or cure diseases that were previously considered untreatable, by correcting the underlying genetic cause.

Gain an understanding of personalized medicine

Personalized medicine tailors treatments to patients’ genetic and individual characteristics, improving effectiveness and reducing side effects, unlike the outdated “one-size-fits-all” approach. It aims for targeted, preventive care, enhancing outcomes and involving patients in their treatment, crucial for rare diseases.

Take your eLearning further

By the end of this course, you will have a better understanding of advanced therapeutic approaches and their potential to revolutionize treatment options for individuals living with rare diseases.

You may also consider enrolling in another course offered by the European Joint Programme on Rare Diseases:

• Diagnosing Rare Diseases: from the Clinic to Research and back
• From Lab to Clinic: Translational Research for Rare Diseases
• Health Data Ethics & Regulatory Frameworks in Rare Disease Research

This course is designed for anyone interested in knowing how recent advances in the medical research field can impact the treatment of rare disorders.

The MOOC will be particularly beneficial for medical students, researchers, patient advocacy groups, healthcare professionals, and biotech developers. However, anyone with a curiosity about science, medicine, and rare conditions is encouraged to participate.

This course has been developed through funding from the EJP RD project.

The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N825575.

• Introduction to innovative therapies and personalized approaches for rare diseases
• Welcome & Introduction
• Setting the scene: the rare diseases landscape
• Personalized innovative therapeutic approaches for rare diseases
• Revision of some fundamental concepts
• Test your knowledge
• Innovative therapies: gene therapies and protein-based approaches
• Welcome & Introduction
• Gene-based therapies: an introduction
• Gene therapy in action
• Gene therapy: new tools and prospects
• Protein-based therapeutics
• Test your knowledge
• Regenerative medicine
• Welcome & Introduction
• Cell therapy approaches
• Transplantation
• Tissue engineering
• Test your knowledge
• Personalized/precision medicine and rare diseases
• Welcome & Introduction
• Introduction to personalized medicine
• The workflow of personalized medicine
• Omics approaches
• Use of omics data in personalized medicine
• Application of personalized medicine to rare diseases
• Test your knowledge
• Personalized therapies and real-world challenges
• Welcome & Introduction
• Personalized therapies in the current approach to innovation
• Patients data use in personalized medicine
• Medicines for small populations: regulatory considerations
• Access to treatments
• Drug economics and personalized medicine
• Personalized medicine: how to protect our innovation?
• Available toolkits and initiatives for rare diseases
• Test your knowledge