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14 August, 2019 - 11:59 By Kate Sweeney

Spirit of Stephen Hawking evoked by Cambridge rare disease summit

Cambridge Rare Disease Network is gearing up for its 4th RAREsummit on September 23 at the Wellcome Genome Campus and excitement is building for what promises to be an innovative event challenging the status quo. 

The organisers don’t do things by halves and from the outset have provided a platform for an impressive collection of hard-hitting speakers from the Cambridge Cluster and University alongside European and US experts.

The late Professor Stephen Hawking, Cambridge’s best-known rare disease advocate, shared his own personal story at the inaugural Cambridge Rare Disease Summit. Professor Hawking was diagnosed with motor neuron disease at 21 and was told science had no answers.

He went on to live a long and productive life with the support of medical and technological advances and Cambridge Rare Disease Summits continue to drill down into how to make this a reality for all 3.5 million Britons affected by rare disease. 

With more than 7000 rare diseases already identified, we each face a one in 17 chance of developing one during our lifetime yet, disturbingly, only five per cent of these have an approved treatment.

Recent advances in genetics, genomic medicine and technologies – such as gene-editing and CRISPR – mean that scientists are better placed than ever before to diagnose, treat and potentially cure rare genetic disorders. 

Likewise, assistive technologies are improving accessibility and independence and Artificial Intelligence is opening up new opportunities for faster and cheaper therapeutic possibilities. 

This year’s RAREsummit will be true to form with a selection of high calibre speakers. Dr Jonathan Milner, co-founder and deputy chairman of Abcam and biotech entrepreneur, will deliver the welcome address.

A keen supporter of CRDN and its ambitions from the outset, he has been involved as a speaker, company sponsor, a significant donor through his foundation The Evolution Education Trust, and most recently joined CRDN’s board of trustees.

Joining Jonathan on stage are Cambridge healthcare pioneers Dr Andy Richards and Dr David Brown, co-founder and chair of rare disease drug-repurposing company Healx. They will share their vision of 21st century digital health where technologies empower patients to manage their own health, find treatments and be connected. 

Local pharmaceutical company representatives Steve Rees of AstraZeneca and Neil Dugdale of Sobi, a pioneering Granta Park based orphan drug company, will join leading rare disease patient advocate Dr Nick Sireau, founder of the AKU Society and Findacure  to explore industry & patients’ current views and future vision of the drug development process. 

Seeking to disrupt the ‘them and us’ relationship dynamic, RAREsummit’s  programme will see CEOs of rare disease advocacy groups such as Emily Crossley of Duchene UK and Dr Paul Wicks from PatientLikeMe, share the stage with industry, academia and healthcare experts to showcase drug development, technology and healthcare projects where collaboration with them has prospered. 

The aim of the agenda is to discover more about rare diseases and for each delegate to take home a toolkit of strategies that can be applied to their own working relationships and projects.

CRDN is a charity formed in 2015 as a strategic hub aimed at accelerating scientific, clinical and social innovation towards effective health care for patients affected by rare diseases.

Co-founded by Dr Tim Guilliams, CEO of Healx, CRDN has the same mission to transform the lives of rare disease patients. 

Guilliams says: “There’s already not enough resources, not enough data yet so many challenges that the only way forward is to collaborate.” 

It is this vision which sparked the idea of developing a network complementary to existing organisations such as One Nucleus, Innovation Forum and Cambridge Network – building an active community of individuals, organisations and companies with the collective capacity to unlock the potential within this vibrant hub of biotech and academic excellence for rare disease awareness, scientific progress and cross-sector collaboration. 

At RAREsummmit19, the strength of this initiative and the power of a such a network to drive change will be apparent. The day will close with CRDN putting its mission into practice during a cross-sector team challenge – an opportunity for all involved to have their say on the future of the UK Rare Disease Strategy.

• For more details and to book tickets visit https://camraredisease.org/summit-2019/

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