Nicolas Sireau

Rare Diseases (eBook, PDF)

Challenges and Opportunities for Social Entrepreneurs

• Format: PDF

Produktbeschreibung

An essential toolkit for social and business entrepreneurs interested in the world of rare diseases, this book presents the latest developments in the field from a global group of experts. An original and timely book, it will help to add knowledge and awareness to a vastly under-published subject.

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Produktdetails

• Verlag: Taylor & Francis eBooks
• Seitenzahl: 278
• Erscheinungstermin: 8. September 2017
• Englisch
• ISBN-13: 9781351278393
• Artikelnr.: 49266391

Autorenporträt

Sireau, Nicolas

Inhaltsangabe

Introduction Nicolas Sireau 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 Nicolas Sireau