Presentation Findacure's co-founder Nick Sireau gave at the Rare Bone Disease Workshop held on 24th June 2014 in Sheffield.

1. Dr Nicolas Sireau, Co-founder and Chairman
An introduction to

6. 7000
Source: Rare Disease UK
rare diseases
of which 80% are very rare
and only 200 have approved treatments

7. Limited
awareness…
Source: Rare Disease UK
…of rare diseases in public and
politics, because each rare
disease struggles by itself

8. Problem 1
Lack of
funding for
rare disease
research

9. The solution?
Many rare
diseases are
fundamental
diseases

10. What is a fundamental
disease?
it’s an extreme and rare disease that
provides insight into common disease
mechanisms

11. statins

12. bisphosphonates

13. neuroacanthocytosis

14. progeria

15. alkaptonuria

16. What makes fundamental
diseases so important?
they prove that rare
diseases are
important
they can unlock our
understanding of
common diseases
1 2

17. Problem 2
Lack of
support for
patients

18. The solution?
Capacity
building for
patient
groups

19. Problem 3
Lack of
commercial
interest

20. The solution?
A non-profit
drug
development
business