The Real Story About Rare and Ultra-Rare Disease from the Ultra Rare Diseases, Disorder & Disabilities Foundation

London, United Kingdom, August 11, 2015 --(PR.com)-- Terence Hoey, CEO of The Ultra Rare Diseases, Disorder & Disabilities Foundation, will be presenting the Case Study entitled “The real story about rare and ultra-rare disease” at the 4th annual Orphan Drugs and Rare Diseases Conference, taking place in London, UK.

In his case study Terence will be presenting a story that needs to be told by people who are living a nightmare with rare and ultra-rare diseases. The Foundation hopes to bring awareness to the audience with a true story of just what it’s like to have a child with an ultra-rare disease: “A story that will explain what difficulties a family goes through...” For the second part of his speech, Terence will give an oversight into how and why the Foundation was put in place.

While the interest for rare diseases has recently grown throughout the entire medical community, the road to an approved orphan drug is a long, challenging and costly one with many challenges on the way including regulatory, financial, sustainability and pricing and reimbursement issues. Through a series of interactive presentations and panel discussions, event attendees will be able to learn how pharmaceutical, biotech, patient organisations and academia are structuring to participate and leverage the growing orphan market.

Featured Speakers for 2015 include:

- Dr Alastair Kent OBE, Director, Genetic Alliance UK, Chair, Rare Disease UK (RDUK)
- Dr Nicolas Sireau, Chairman & CEO, AKU Society
- Dr Tim Miller, President & CEO, Abeona Therapeutics
- Dr Anne Marquet, Principal Clinical Scientist, Rare Diseases, Roche Pharma Research and Early Development
- Christine Lavary, Chief Executive, MPS Society UK
- Dr Carlos R. Camozzi, Chief Medical Officer, Orphazyme
- Michael Skynner, Head of Rare Disease External Alliances, Pfizer
- David Boothe, Global Commercial Leader – GSK Rare Diseases, GSK
- Dr Didier Caizergues, Head of regulatory Affairs Department, International Regulatory Affairs department, GENETHON
- Stephane Demotz, Founder, DORPHAN S.A.

The event programme also includes two interactive half-day post-conference workshops, entitled Market access to orphan drugs: Controversies, trends and solutions (Led by Colette Hamilton, Managing Director, ATP Market Access) and The rare disease patient perspective-from regulatory to clinical execution (Led by Christa van Kan, Team Lead Clinical Execution, PSR Orphan Experts, Steve Phinder, PhD Director, Envestia Ltd, and Nicolas Sireau, PhD, Chairman and CEO, AKU Society)

About SMi Group:
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