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From Lab to Clinic: Translational Research for Rare Diseases

Gain an overview of the issues, challenges, and opportunities in translating research into treatments for rare disease patients.

2,057 enrolled on this course

A close up shot of a female scientist conduction clinical research, as she looks down a microscope, studying rare diseases

From Lab to Clinic: Translational Research for Rare Diseases

2,057 enrolled on this course

  • 5 weeks

  • 4 hours per week

  • Digital certificate when eligible

  • Introductory level

Find out more about how to join this course

Discover how research is translated into drugs and treatments for RD patients

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

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

Describe translational medicine phases like drug discovery, clinical trials and regulatory approval

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 the drug development process, gaining contextual insights into this vital area of medical research.

Understand the do’s and don’ts when moving 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.

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Skip to 0 minutes and 0 seconds Developing therapies for rare diseases is complex. Still, you have the ambition to contribute to this as a PhD student, a health care professional, a researcher, a biotech developer, a patient, or a patient’s representative, and you want to learn the different steps involved in this process. The European Joint Programme on Rare Diseases

Skip to 0 minutes and 22 seconds has developed the online course “From Lab to clinic: translational research for rare diseases”, where you can discover the issues, challenges, and opportunities in translating basic research findings into medical treatments and therapies for rare diseases. Five weeks of courses, 15 hours of content, 117 learning steps, 47 videos, nine international experts, including patient experts, and lots of exercises, quizzes, and activities. Are you ready to learn more about translational research for rare diseases? Are you ready to make a change for all the patients? We’re counting on you.

What topics will you cover?

  • 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

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...

  • 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

Who is the course for?

This course is designed for researchers and students in medicine and health-related research fields, as well as healthcare professionals wanting to further their knowledge of translational research in rare diseases.

It is also suitable for biotech and start-up developers and Patients Advocacy Organisation representatives who want to increase their understanding of current practices in therapeutic developments for rare diseases.

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?

I have worked in preclinical therapy development in rare diseases (mostly Duchenne muscular dystrophy) for over 20 years and am also involved in multistakeholder networking and education.

I am a board-certified neurologist and neuropathologist with long-standing experience in neuromuscular diseases. I coordinate the Europeann reference network for rare neuromuscular diseases (EURO-NMD)

I am a Research Policy and Initiatives Director at EURORDIS, Rare Diseases Europe. Since 2019, I have been involved in the development of several MOOCs as part of the EJP RD.

Innovation Manager in the European Joint Programme on Rare Diseases and National Coordinator EATRIS Norway. PhD Molecular Biology on Rare Disease. 10 years of Drug discovery experience from industry.

Agustín Arasanz is a biologist and works as an Innovation Manager at VHIR, Barcelona. In 2019 he joined EJP -RD to coordinate the Mentoring service for Rare diseases research project.

I am Head of Training at EATRIS, the European Research Infrastructure for Translational Medicine. I coordinated the development of week 1 within the European Joint Programme for Rare Diseases.

PhD in Genetics and Postdoc experience; working for the past years in the management of EU projects for rare diseases. In Jan 2021, I joined the Coordination of ERN EURO-NMD as Senior Project Manager.

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. The first course will address research issues related to diagnosis in the rare diseases context.

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!

Subscribe & save


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

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

Limited access


Sample the course materials

  • Access expires 24 May 2024

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

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  • Learn through a mix of bite-sized videos, long- and short-form articles, audio, and practical activities
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  • Complete 90% of course steps and all of the assessments to earn your certificate

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