| ABOUT THE EDITOR
Dr Nicolas Sireau is Chairman of the AKU Society, a medical charity that works to find a cure for and support patients with AKU, which affects his two sons. The AKU Society is a fast-growing patient movement, with formal patient groups in the UK, France, Italy, Germany, the Netherlands and North America. It is spearheading an international consortium of biotechs, pharma companies, universities, hospitals and clinical trial centres across Europe, the Middle East, Asia and North America. He is the Co-founder and Chairman of Findacure, a new charity that raises awareness and funds research into fundamental diseases: extreme and exceptional diseases that advance our understanding of medicine and help us discover potential new treatments. Dr Sireau is also a former non-executive Director of GenSeq, a bioinformatics company. He is a fellow of the Ashoka fellowship of social entrepreneurs and of the Royal Society of Arts. Dr Sireau's previous career was in international development, where he set up SolarAid, an award-winning social enterprise bringing solar power to Africa, and wrote several books on international aid. Dr Sireau has a PhD in Social Psychology, an MSC in Business Management, an MA in Journalism Studies, and an MA (Oxon) in History and Economics.
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| | Latest developments in the world of rare disease entrepreneurship from a global group of experts.
We are pleased to announce the publication of Rare Diseases Challenges and Opportunities for Social Entrepreneurs.Order your copy direct from Greenleaf Publishing and receive 20% discount.
List price: £25.00 | €32.00 | $45.00
Online price: £20.00 | €25.60 | $36.00 (not including postage and packing). Read samples (PDF): Introduction | Conclusion |
| Rare Diseases
Challenges and Opportunities for Social Entrepreneurs Edited by Nicolas Sireau
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. |
| Contents
Introduction
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
Conclusion |
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