Advancing treatments for rare diseases Together
  • Our model

    With patients, we address the translational gap in rare disease research and development and bridge academic research efforts with pharmaceutical development
  • First programme

    EspeRare investigates the therapeutic potential of Rimeporide in Duchenne Muscular Dystrophy, a lethal and pediatric rare disease
  • Our mission

    The Foundation focuses on overcoming roadblocks that prevent new therapies to reach rare diseases patients

Our mission and vision

In collaboration with patient groups and other key stakeholders, we uncover the potential of existing pharmacotherapies to address severe therapeutic unmet needs in rare diseases.

Our model

We strive to apply our patient-centric model and our pharma R&D expertise to advance the discovery of new treatments for these underserved patients.

R&D programmes

EspeRare aims to build a diversified portfolio of programmes with a strong potential to treat rare diseases. Our first programme is in Duchenne Muscular Dytrophy.

Recent news

The last advances of the Rimeporide clinical study have been presented at the 22nd World Muscle Society congress , in St Malo. Numerous...
The Food and Drug Administration (FDA), theĀ  US Department of Health and Human Services, has granted an Orphan Drug Designation (ODD) for...


  • It is time to renovate and accelerate the discovery and development of drugs for rare diseases. Esperare is an essential player in the innovative, collaborative system that will be created to make a difference in this world.
    Sharon Terry
    President of EspeRare and CEO of Genetic Alliance
  • As a nonprofit organisation, our priorities are not determined by the size of a market, they are solely defined by the medical needs and great science. Above all, we strive to apply our patient-centric model and pharma know-how to advance new treatments for underserved patients.
    Caroline Kant
    EspeRare's Founder and Executive Director
  • For me it is a privilege to represent Merck Serono on the Board of EspeRare, I firmly believe that this exciting new Foundation can make a real difference to the lives of patients with Duchenne Muscular Dystrophy and other rare diseases.
    Ewen Sedman
    Board member
  • Fostering access to health for patients that are the most in need is what this foundation is about, and is what I am about.
    Beatrice Greco
    EspeRare's founder and member of the Board
  • Duchenne is a heartbreaking disease. Children like Laurent, my cousin, affected with this disease are bright and engaged but as they grow up, they inexorably get weaker and experience the loss of the few abilities they had acquired. I have in my genes the eagerness to find treatments for this disease and I am committed to give them the strength to fight their disease.
    Florence Porte
    EspeRare's founder and R&D Director