Presentation at Rare Bone Disease Workshop - 24th June 2014
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Transcript of Presentation at Rare Bone Disease Workshop - 24th June 2014
Dr Nicolas Sireau, Co-founder and Chairman
An introduction to
7000
Source: Rare Disease UK
rare diseases of which 80% are very rare
and only 200 have approved treatments
Limited awareness…
Source: Rare Disease UK
…of rare diseases in public and politics, because each rare disease struggles by itself
Problem 1
Lack of funding for rare disease
research
The solution?
Many rare diseases are fundamental
diseases
What is a fundamental disease?
it’s an extreme and rare disease that provides insight into common disease
mechanisms
statins
bisphosphonates
neuroacanthocytosis
progeria
alkaptonuria
What makes fundamental diseases so important?
they prove that rare diseases are important
they can unlock our understanding of common diseases
1 2
Problem 2Lack of
support for patients
The solution?
Capacity building for
patient groups
Problem 3Lack of
commercial interest
The solution?
A non-profit drug
development business