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Innovative Therapies and Personalised Medicine for Rare Diseases

Learn how recent advances in medicine research are transforming the treatment of rare diseases.

474 enrolled on this course

Female scientists working in a lab near a microscope. On the right: some representations of the double helix

Innovative Therapies and Personalised Medicine for Rare Diseases

474 enrolled on this course

  • 5 weeks

  • 4 hours per week

  • Digital certificate when eligible

  • Intermediate level

Find out more about how to join this course

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to patients’ genetic and individual characteristics, improving effectiveness and reducing side effects, unlike the outdated “one-size-fits-all” approach.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:

Syllabus

  • Week 1

    Introduction to innovative therapies and personalized approaches for rare diseases

    • Welcome & Introduction

      Welcome to this MOOC! In this subactivity you will be introduced to this course, by understanding in which context it has been developed, who are the authors and the main subjects that will be addressed throughout the weeks.

    • Setting the scene: the rare diseases landscape

      What is a rare disease, what implies living with a rare disease? In this subactivity we will outline these topics and we will describe the most relevant initiatives developed in the RD landscape.

    • Personalized innovative therapeutic approaches for rare diseases

      What is meant by innovative therapies? And personalized medicine? How can these approaches help in the treatment and care of rare diseases? Let's find out in the next subactivity.

    • Revision of some fundamental concepts

      This section will review essential concepts that are crucial to fully comprehend the upcoming weeks.

    • Test your knowledge

      What have you learnt and understood this week? Test your knowledge with a multiple choice quiz and reflect on the week's content

  • Week 2

    Innovative therapies: gene therapies and protein-based approaches

    • Welcome & Introduction

      Welcome to week2! This week we will learn about gene-based therapies and protein-based therapeutics.

    • Gene-based therapies: an introduction

      Gene-based therapies involve using genetic material to treat or prevent diseases. This can include gene editing, gene replacement, or gene modulation to address genetic disorders and other illnesses.

    • Gene therapy in action

      In this subactivity, we will explore how a gene therapy is concretely planned, developed and tested and we will understand how it can impact the life of a family of a person living with a RD.

    • Gene therapy: new tools and prospects

      Genome editing approaches use tools like CRISPR to modify an organism's DNA. This can involve adding, removing, or altering specific DNA sequences to study gene function or treat genetic diseases.

    • Protein-based therapeutics

      Protein-based therapies show promise for diverse diseases. Antibody-based and protein replacement therapies are key categories, offering unique approaches for treatment.

    • Test your knowledge

      What have you learnt and understood this week? Test your knowledge with a multiple choice quiz and reflect on the week's content.

  • Week 3

    Regenerative medicine

    • Welcome & Introduction

      This week, we delve into regenerative medicine, cell-based methods, transplantation, and tissue engineering, exploring definitions, historical significance, and latest advancements in these cutting-edge fields.

    • Cell therapy approaches

      Cell-based therapy approaches are therapeutic approaches that involve the use of living cells such as stem cells, immune cells or differentiated cells to treat or to prevent diseases.

    • Transplantation

      By replacing damaged or malfunctioning organs with healthy ones from donors, organ transplants offer a transformative solution for individuals suffering from various debilitating conditions.

    • Tissue engineering

      Tissue engineering is a multidisciplinary field aiming to create functional three-dimensional tissues to restore, maintain, or improve tissue function using a combination of cells, engineering, and materials.

    • Test your knowledge

      What have you learnt and understood this week? Test your knowledge with a multiple choice quiz and reflect on the week's content.

  • Week 4

    Personalized/precision medicine and rare diseases

    • Welcome & Introduction

      Welcome to week 4! This week will focus on personalized medicine and its application to rare diseases.

    • Introduction to personalized medicine

      Here we explore the definitions of personalized and precision medicine. You will also get introduced to the concepts of biomarker, biosamples and biobanks.

    • The workflow of personalized medicine

      A personalized medicine approach involves collecting patient data, analyzing it to identify biomarkers, developing tailored treatment plans, monitoring patient response, and adjusting treatments as needed to optimize effectiveness.

    • Omics approaches

      Omics approaches study the roles and interactions of various cellular molecules. They enable comprehensive molecular profiling, leading to precise diagnoses and targeted therapies.

    • Use of omics data in personalized medicine

      Here we explore how omics data is translated into meaningful observations through data standardization and processing. The role of AI will also be discussed.

    • Application of personalized medicine to rare diseases

      In this subactivity we will illustrate some concrete examples of applications of personalized medicine for rare diseases.

    • Test your knowledge

      What have you learnt and understood this week? Test your knowledge with a multiple choice quiz and reflect on the week's content.

  • Week 5

    Personalized therapies and real-world challenges

    • Welcome & Introduction

      Here we are at the end of our journey. We hope you find this course interesting for you. Prior to concluding, we will delve into real-world challenges associated with the implementation of innovative and personalized therapies.

    • Personalized therapies in the current approach to innovation

      Here we discuss the importance of stakeholder collaboration in personalized medicine to address disparities in healthcare systems and the need for harmonized strategies. We introduce also exisiting initiatives and partnerships.

    • Patients data use in personalized medicine

      What is the role of health data in rare disease research, and which framework governs its use?

    • Medicines for small populations: regulatory considerations

      Let's explore how personalized approaches and innovative therapies are considered from a regulatory perspective, introducing the concept of orphan drugs. We will then see the current incentives for the development of these drugs.

    • Access to treatments

      Here we address the need for equitable access to treatments for all patients, emphasizing international and EU efforts to support this, particularly for rare diseases.

    • Drug economics and personalized medicine

      Here, we explore the economic and regulatory challenges in drug development, particularly in the context of rare diseases, and propose possible solutions as crowdfunding as a funding model.

    • Personalized medicine: how to protect our innovation?

      How intellectual property (IP) is handled in the context of academics and the pharmaceutical industry?

    • Available toolkits and initiatives for rare diseases

      As we come to the end of the week and the course, we would like to provide you with a list of tools and initiatives that can help speed up the drug development process, with a focus on rare diseases.

    • Test your knowledge

      What have you learnt and understood this week? Test your knowledge with a multiple choice quiz and reflect on the week's content.

When would you like to start?

Start straight away and join a global classroom of learners. If the course hasn’t started yet you’ll see the future date listed below.

  • Available now

Learning on this course

On every step of the course you can meet other learners, share your ideas and join in with active discussions in the comments.

What will you achieve?

By the end of the course, you‘ll be able to...

  • Describe what is meant by rare diseases and illustrate the challenges related to the treatment of such a kind of diseases
  • Explain what the most innovative approaches are for developing therapies, such as gene therapy, protein-based approaches, and cell therapy
  • Describe what is meant by regenerative medicine and how cell therapy, tissue engineering and transplants contribute to this field
  • Summarise why all these approaches are particularly relevant for rare diseases
  • Debate what personalized medicine is and explain how it differs from the classical “one fits all approach”
  • Describe the role of clinical data in developing innovative personalized treatments
  • Describe how health data are optimally managed from the regulatory perspective
  • Discuss the current real-world challenges related to the development of personalized treatments and how they could be overcome

Who is the course for?

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.

EU logo

Who will you learn with?

Head of Immunology at La Paz University Hospital. Director of a group at the Spanish Rare Disease Network Research Centre (CIBERER). Coordinator of the Research WG at the ERN-TRANSPLANTChild.

Giovanni Migliaccio is the Scientific Director of CVBF and a member of the European Paediatric Translational Research Infrastructure (EPTRI) Board of Directors. He is an expert in the area of ATMPs.

I am an Immunology Specialist at La Paz University Hospital. I am responsible for the Transplant Immunology Section, manage quality and research at IdiPAZ, also collaborating with ERN-TransplantChild

I'm Health Research Project Manager at CVBF. I'm involved in several national and international projects. Since 2023, I have been involved in the development of the MOOCs as part of the EJP RD.

Scientific projet manager at Fondation Maladies Rares. I coordinate the development of this and other MOOCs within the European Joint Programme on Rare Diseases.

Who developed the course?

Foundation for Rare Diseases

The Foundation for Rare Diseases is coordinating a series of courses on rare diseases research topics within the context of the European Joint Programme on Rare Diseases.

European Joint Programme on Rare Diseases

The European Joint Programme on Rare Diseases (EJP RD) brings over 130 institutions (including all 24 ERNs) from 35 countries to create a comprehensive, sustainable ecosystem allowing a virtuous circle between research, care and medical innovation.

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Ways to learn

Choose the best way to learn for you!

Buy this course

$134/one-off payment

Fulfill your current learning need

  • Access to this course
  • Learn at your own pace
  • Discuss your learning in comments
  • Printed and digital certificate when you’re eligible

Subscribe & save

$349.99 for one year

Automatically renews

Develop skills to further your career

  • Access to this course
  • Access to 1,000+ courses
  • Learn at your own pace
  • Discuss your learning in comments
  • Digital certificate when you're eligible

Cancel for free anytime

Limited access

Free

Sample the course materials

  • Access expires 23 Aug 2024

Find out more about certificates, Unlimited or buying a course (Upgrades)

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