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