Rare diseases : challenges and opportunities for social entrepreneurs / edited by Nicolas Sireau.

Includes bibliographical references.

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. The book will be an essential toolkit for social and business entrepreneurs who are interested in the world of rare/orphan diseases. It has the rigour of an academic publication, along with the clarity of a lay publication. An original and timely book, Rare Diseases will help to add knowledge and awareness to a vastly under-published subject.

Print version record.

Cover; Half title; Title; Copyright; Contents; Introduction; 1Toward a focused, multinational, rare disease awareness initiative; 2The challenges of scaling up an orphan drug enterprise; 3Building an entrepreneurial patient movement; 4The practicalities of clinical development of drugs for rare diseases; 5Navigating orphan drugs through the regulatory maze; 6Drug repositioning strategies for rare and orphan diseases; 7Why patient registries are crucial for finding cures for rare diseases.

8 Challenges, strategies and lessons learned for the setting up and running of a European Reference Network for rare disease9Managing research advances into a rare disease; 10The BLACKSWAN Foundation for rare diseases; 11The rise and fall of Sanfilippo syndrome; 12Lobbying for a national rare disease plan in the UK; 13The global drug development process; Conclusion; Index; Back cover.

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