Rare Diseases: Challenges and Opportunities for Social Entrepreneurs

In Clinical Trials, Partnering & Investment, Regulation and Compliance by Matt TurnerLeave a Comment

rare diseases, orphan drugs, Nicolas SireauLatest developments in the world of rare disease entrepreneurship from a global group of experts.

Just Announced: The forthcoming publication of Rare Diseases Challenges and Opportunities for Social Entrepreneurs on 5 April 2013. Edited by Dr. Nicolas Sireau, Chairman of the AKU Society, the new publication contains chapters from leading players in the rare disease space, such as Prosensa, NORD, Rare Disease UK, Lysogene, Orphan Europe and Genetic Alliance.

There are 7,000 rare diseases affecting 6%-8% of the global population. That’s 3.5 million people in the UK alone. Yet only 200 rare diseases have approved treatments. In recent years, there has been a surge of interest from business and social entrepreneurs in the field of health – including looking at ways to treat rare disease patients better and faster. This book presents some of the latest developments in the world of rare disease entrepreneurship from a global group of experts. It examines the topic from the business angle, considering the drug development process and providing case studies of successful orphan drug enterprises.

It also looks at rare diseases from the perspective of the patient, analysing the growing rare disease patient movement, a successful patient group that uses social enterprise techniques, and chapters on key requirements for helping patients with rare diseases through registries and centres of excellence.

Pre-order your copy now and receive 30% discount.

Contents by Chapter

1 Toward a focused, multinational, rare disease awareness initiative
Peter Saltonstall and E. Michael D. Scott
2 The challenges of scaling up an orphan drug enterprise
Hans Schikan
3 Building an entrepreneurial patient movement: A global case study from the AKU Society
Oliver Timmis
4 The practicalities of clinical development of drugs for rare diseases
Anthony K Hall
5 Navigating orphan drugs through the regulatory maze: Successes, failures and lessons learned
Remco de Vrueh, Harald Heemstra and Michelle Putzeist
6 Drug repositioning strategies for rare and orphan diseases: A cost-effective approach of new uses for existing drugs
Maria P. del Castillo-Frias, Andrew J. Doig and Farid Khan
7 Why patient registries are crucial for finding cures for rare diseases
Pat Furlong and Kyle Brown
8 Challenges, strategies and lessons learned for the setting up and running of a European Reference Network for rare disease
Samantha Parker and Stephen Lynn
9 Managing research advances into a rare disease: Case study of the Myrovlytis Trust
John Solly and Galina Shyndriayeva
10 The BLACKSWAN Foundation for rare diseases
Olivier Menzel and Dr Silvia Panigone
11 The Rise and fall of Sanfilippo syndrome
Karen Aiach
12 Lobbying for a national rare disease plan in the UK: Lessons for rare disease patient alliances
Stephen Nutt
13 The global drug development process: What are the implications for rare diseases and where must we go?
Sharon F. Terry with Jayson Swanson

On behalf on Nicolas, I am delighted to inform that all royalties from book sales will be donated to rare disease patient groups so make sure you share this blog and Pre-order your copy now.

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