An introduction to Dr Nicolas Sireau, Co-founder and Chairman 7000 - - PowerPoint PPT Presentation

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Jul 25, 2023 289 likes •602 views

An introduction to Dr Nicolas Sireau, Co-founder and Chairman 7000 rare diseases of which 80% are very rare and only 200 have approved treatments Source: Rare Disease UK 3.5 million people affected in the UK, hundreds of millions worldwide

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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

f which 80% are very rare

and only 200 have approved treatments

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3.5 million

Source: Rare Disease UK

people affected in the UK, hundreds of millions worldwide

Source: Rare Disease UK

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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

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Problem 2

Lack of support for patients

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The solution?

Capacity building for patient groups

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Problem 3

Lack of commercial interest

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The solution?

A non-profit drug development business

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A hybrid social enterprise structure

Findacure Partnership: the charity Findacure Development: the non-profit business

OWNS

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A focused implementation strategy

Campaigning and education Fundraising and marketing Scientific research and drug development Patient group support

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The board and staff

Board:

 Dr Nicolas Sireau, social entrepreneur and father of two

children with a rare disease

 Dr Anthony Hall, medical doctor and orphan drug

entrepreneur

 Andy Milligan, global branding expert  Edna Kissman, owner of communications business  Dr John Solly, geneticist and charity CEO  Julie Walters, founder of healthcare PR company

Staff:

 Flóra Raffai, Project Manager and LSE graduate

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The Scientific Advisory Board

 Prof Jim Gallagher, Univ of Liverpool  Prof L. Ranganath, Royal Liverpool Univ Hospital  Prof Jonathan Jarvis, Liverpool John Moore Univ  Dr Rob Forsyth, Newcastle Univ  Dr Anil Mehta, Dundee Univ  Dr Farid Khan, Protein Technologies  Dr Nathalie Kayadjanian, rare disease consultant  Dr Andrew R. Tee, Cardiff Univ  Oliver Timmis, AKU Society

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Launches 10th February 47 day campaign on Indiegogo Aim: Raise $25,000 to continue patient workshops and build

nline toolkit for patient groups

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First Scientific Workshop on Fundamental Diseases Sidney Sussex College, Cambridge CB2 3HU Friday 14 March 1.30pm-5.30pm

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Rare Diseases: Challenges and Opportunities for Social Entrepreneurs

A new book

ut now!

With chapters from leaders in the rare disease sector Contact: nick@akusociety .org

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