Advancing treatments for rare diseases together

Addressing rare diseases

Rare diseases are a major healthcare burden 

8 to 10% of the global population is affected by rare diseases; it includes diseases that impact fewer than 1 in 2,000 people. Unfortunately,  only 5% of  rare diseases (also called "orphan diseases") have approved treatments.

Most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases will not live to celebrate their fifth birthday.

Over +7’000 rare diseases have been identified; They affect 1 out of 10 people in Europe and in the United States; Only ~5 % have approved therapeutic solutions; 80% are of genetic origin and 50% of rare disease patients are children

 

An opportunity to address a major gap in the medical landscape  affecting children

Several therapeutic opportunities to treat rare disease patients exist but are too often left in “drawers” of pharmaceutical companies or universities. These opportunities are often never tested nor developed because biopharmaceutical companies are rarely willing to risk investing Research and Development (R&D) budget for this small market and lower financial return potential. Academia on the other hand often lack the know-how to conduct robust drug development especially in late phases of clinical development.  

New hope for patients with life-threatening rare diseases

Developing new treatments is expensive, very lengthy and requires tight coordination between a large spectrum of research and development (R&D) activities and expertise. Therefore, EspeRare focuses on accelerating and increasing the chance of success of discovering and developing novel treatments for rare diseases by repositioning existing therapies.

Patients are at the core of healthcare progress in rare diseases

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  • "As a not-for-profit organisation, our priorities are defined by the unmet medical needs of rare diseases patients, the wealth of good science available, and the therapeutic potential of the treatment we develop. We aren’t driven by commercial interests or the size of a market. Above all, we strive to apply our patient-centric model and our pharma R&D expertise to advance the discovery of new, life-changing treatments for these underserved patients".
  • Caroline Kant
  • Founder and Executive Director

Useful links

 

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