If you are experiencing problems downloading PDF or HTML fulltext, our helpdesk recommend clearing your browser cache and trying again. If you need help in clearing your cache, please click here . Still need help? Email help@ingentaconnect.com

Rare Diseases: Challenges and Opportunities for Social Entrepreneurs
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.

Publisher: Greenleaf Publishing in association with GSE Research

More about this publication?
Related content

Favourites:Add to Favourites

Free Content Preliminary Material
pp. i-vi(6)
Author: Sireau, Nicolas

Favourites:Add to Favourites

Introduction
pp. 1-6(6)
Author: Sireau, Nicolas

Favourites:Add to Favourites

Toward a focused, multinational, rare disease awareness initiative
pp. 7-21(15)
Authors: Saltonstall, Peter; Scott, E. Michael D.

Favourites:Add to Favourites

The challenges of scaling up an orphan drug enterprise
pp. 22-42(21)
Author: Schikan, Hans

Favourites:Add to Favourites
Favourites:Add to Favourites

Navigating orphan drugs through the regulatory maze: Successes, failures and lessons learned
pp. 87-113(27)
Authors: de Vrueh, Remco; Heemstra, Harald; Putzeist, Michelle

Favourites:Add to Favourites

Drug repositioning strategies for rare and orphan diseases: A cost-effective approach for new uses for existing drugs
pp. 114-149(36)
Authors: del Castillo-Frias, Maria P.; Doig, Andrew J.; Khan, Farid

Favourites:Add to Favourites

Why patient registries are crucial for finding cures for rare diseases
pp. 150-158(9)
Authors: Furlong, Pat; Brown, Kyle

Favourites:Add to Favourites

Managing research advances into a rare disease: Case study of the Myrovlytis Trust
pp. 176-192(17)
Authors: Solly, John; Shyndriayeva, Galina.

Favourites:Add to Favourites

The BLACKSWAN Foundation for rare diseases
pp. 193-200(8)
Authors: Menzel, Olivier; Panigone, Silvia

Favourites:Add to Favourites

The rise and fall of Sanfilippo syndrome
pp. 201-206(6)
Author: Aiach, Karen

Favourites:Add to Favourites
Favourites:Add to Favourites

Free Content Supplementary Material
pp. 263-272(10)
Author: Sireau, Nicolas

Share Content

Access Key

Free Content
Free content
New Content
New content
Open Access Content
Open access content
Subscribed Content
Subscribed content
Free Trial Content
Free trial content
Cookie Policy
X
Cookie Policy
ingentaconnect website makes use of cookies so as to keep track of data that you have filled in. I am Happy with this Find out more