Advancing treatments for rare diseases Together

Advancing treatments

EspeRare's model to advance treatments in rare diseases:

With patients groups, we drive translational research and development activities to advance treatments for rare diseases by:

  1.  Addressing high unmet needs in rare diseases by iditenfying existing abandoned therapies, thanks to our experts network and our proprietary informatics platform.

  2. Applying our pharmaceutical expertise to develop our selected programmes until we establish clinical proof of concept in patients.
  3. Secure partners for full development and commercialisation, therefore opening a clear path forward for the medicine to reach patients.

EspeRare Foundation bridges the gap between academic research and drug development

EspeRare addresses the translational gap: using our pharma-know, we address the translational “death valley” in rare disease research and development by bridging academic research efforts with pharmaceutical development, thus overcoming the roadblocks that prevent new therapies from reaching rare disease patients.


What is translational research?

Translational research applies findings from basic science to enhance human health. In a medical research context, it aims to "translate" findings in fundamental research into medical practice and meaningful health outcomes.