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

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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

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 Source: Rare Disease UK Source: Rare Disease UK
Limited awareness… …of rare diseases in public and politics, because each rare disease struggles by itself Source: Rare Disease UK
Lack of funding for Problem 1 rare disease research
Many rare diseases are The solution? 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? 1 2 they can unlock our they prove that rare understanding of diseases are common diseases important
Lack of support for Problem 2 patients
Capacity building for The solution? patient groups
Lack of commercial Problem 3 interest
A non-profit drug The solution? development business
A hybrid social enterprise structure Findacure Partnership: the charity OWNS Findacure Development: the non-profit business
A focused implementation strategy Fundraising Campaigning and marketing and education Scientific research Patient group and drug support development
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
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
Launches 10 th February 47 day campaign on Indiegogo Aim: Raise $25,000 to continue patient workshops and build online toolkit for patient groups
First Scientific Workshop on Fundamental Diseases Sidney Sussex College, Cambridge CB2 3HU Friday 14 March 1.30pm-5.30pm
Rare Diseases: Challenges and Opportunities for Social Entrepreneurs A new book out now! With chapters from leaders in the rare disease sector Contact: nick@akusociety .org

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