Learn more about rare diseases!

The Foundation for Rare Diseases has developed a series of three online courses with European and international partners to better understand innovative therapies, health data, and translational research in the field of rare diseases.

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

Developed with the support of a medical education grant from ASGCT and Pfizer, this course focuses on the scientific and translational foundations of gene and gene‑modified cell therapies for rare diseases.

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

Co‑developed within the European Joint Programme on Rare Diseases (EJP RD), this course addresses the role of health data in rare disease research, including ethical issues, regulatory frameworks, data sharing, and stakeholder perspectives.

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

Also co‑developed within EJP RD, this course follows the journey from discovery to treatment: drug development, clinical trials, regulatory approval, and the specific challenges of rare disease research.

These courses are aimed at students, researchers, healthcare professionals, biotech and start‑up developers, patient representatives, and anyone interested in rare diseases and biomedical research.
Additional MOOCs developed by the Foundation for Rare Diseases and its partners are also available on FutureLearn: https://www.futurelearn.com/partners/french-foundation-rare-diseases