I started by defining what a rare disease is and stressing the specific issues linked to its rarity.

We will then discover the landscape of translational medicine by defining what is meant by “translation” and by introducing the typical process of developing therapies and highlighting the specific challenges and opportunities for rare diseases.

The main actors of the rare disease landscape will be presented, with a particular focus on the stakeholders and contributors of the development of orphan drugs.

A case study will illustrate the specific challenges but also opportunities encountered when developing a treatment for a rare disease.