Advancing treatments for rare diseases Together

Advancing treatments

The EspeRare 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. Discovering therapeutic value of existing pharmacotherapies to address high unmet needs in rare diseases by capitalizing on our expert networks and our knowledge management approach.
  2. Applying our pharmaceutical expertise to our selected programmes until we establish clinical proof of concept in patients.
  3. Thriving to secure partners for full development and commercialisation, therefore opening a clear path forward for the medicine to reach patients.

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.

About translational research

With its focus on removing barriers to multi-disciplinary collaboration, translational research has the potential to drive the translation of basic research into applied science and ultimately allow more treatments to reach patients.
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