Short online courses on rare disease research developed by Foundation for Rare Diseases

The Foundation for Rare Diseases is a French non-profit organisation based in Paris, dedicated to advancing research and knowledge in the field of rare diseases. It is offering three MOOCs on rare disease research that may be of interest.  You will find the details of each course below:

1. MOOC From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases

  • Overview: Funded through an ASGCT & Pfizer Medical Education Grant, this course covers the scientific and translational foundations of gene therapy—including non-viral delivery, viral vectors, gene editing, RNA therapeutics, and gene-modified hematopoietic stem cell therapy for blood disorders. It highlights how these therapies are opening new treatment possibilities for rare diseases. Co-developed with leading US and European researchers, it also features insights from patients who have received gene therapy.
  • Link to enroll: https://www.futurelearn.com/courses/from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases
  • Access: Free for 3 weeks after enrollment 
  • Expert Support: Experts in the field are online to answer questions until May 8
  • Target Audience: University students, early-career researchers in biology, life sciences, and medicine, as well as anyone interested in learning about gene and cell therapies for rare diseases 
  • Accreditation: CPD-certified 

2. EJPRD MOOC Health Data Ethics & Regulatory Frameworks in Rare Disease Research 

  • Overview: Co-developed by ERN Epicare, EURORDIS, Fondazione Gianni Benzi, and FFRD in the context of the EJPRD (European Joint Programme on Rare Diseases), this course explores the role of health data in rare disease research. It covers the types of research data involved, why health data matters, key stakeholders, current challenges and possible solutions, the benefits of data sharing, ethical considerations, regulatory frameworks, available resources, and stakeholder perspectives.
  • Link to enroll: https://www.futurelearn.com/courses/health-data-ethics-and-regulatory-frameworks-in-rare-disease-research
  • Access: Free for 4 weeks after enrollment 
  • Expert Support: Experts are online to answer questions until May 15
  • Target Audience: Undergraduates, PhD students, healthcare professionals, researchers, patient advocacy representatives and anyone interested in health data and rare diseases.
  • Accreditation: CPD-certified 

3. EJPRD MOOC From Lab to Clinic: Translational Research for Rare Diseases 

  • Overview: Co-developed by ERN EuroNMD, EATRIS, EURORDIS, LUMC, and FFRD in the context of the EJPRD (European Joint Programme on Rare Diseases), this course follows the journey from discovery to treatment, explaining how medicines are developed, tested in clinical trials, and approved. It also highlights key issues in rare disease drug development, including drug repurposing, patient-reported outcomes, and patient involvement in preclinical and clinical research.
  • Link to enroll: https://www.futurelearn.com/courses/introduction-to-translational-research-for-rare-diseases
  • Access: Free for 5 weeks after enrollment 
  • Expert Support: Experts are online from April 27 to June 13
  • Target Audience: Researchers, students in medicine and health-related fields, healthcare professionals, biotech and start-up developers, and patient advocacy representatives 
  • Accreditation: CPD-certified