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...
Gespeichert in:
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| Format: | Elektronisch E-Book |
| Sprache: | English |
| Veröffentlicht: |
Sheffield, England :
Greenleaf Publishing,
2013.
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| Schlagworte: | |
| Online-Zugang: | Volltext |
| Zusammenfassung: | 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. |
| Beschreibung: | 1 online resource (vi, 264 pages) : illustrations |
| Bibliographie: | Includes bibliographical references. |
| ISBN: | 9781909493209 1909493201 9781906093525 1906093520 9781351278409 1351278401 |
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| 245 | 0 | 0 | |a Rare diseases : |b challenges and opportunities for social entrepreneurs / |c edited by Nicolas Sireau. |
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| 520 | 8 | |a 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. | |
| 588 | 0 | |a Print version record. | |
| 505 | 0 | |a 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. | |
| 505 | 8 | |a 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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| 650 | 2 | 2 | |a Entrepreneurship |x organization & administration |0 https://id.nlm.nih.gov/mesh/D016520Q000458 |
| 650 | 2 | 2 | |a Orphan Drug Production |x methods |0 https://id.nlm.nih.gov/mesh/D009965Q000379 |
| 651 | 2 | |a Europe |0 https://id.nlm.nih.gov/mesh/D005060 | |
| 650 | 2 | |a Drug Industry |0 https://id.nlm.nih.gov/mesh/D004345 | |
| 650 | 6 | |a Maladies rares. | |
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| contents | 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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| discipline | Medizin |
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| geographic | Europe. http://id.loc.gov/authorities/subjects/sh85045631 Europe https://id.nlm.nih.gov/mesh/D005060 Europe. Europe fast https://id.oclc.org/worldcat/entity/E39PBJxCxPbbk4CPJDQJb4r6rq |
| geographic_facet | Europe. Europe |
| id | ZDB-4-EBA-ocn862577543 |
| illustrated | Illustrated |
| indexdate | 2024-11-27T13:25:37Z |
| institution | BVB |
| isbn | 9781909493209 1909493201 9781906093525 1906093520 9781351278409 1351278401 |
| language | English |
| oclc_num | 862577543 |
| open_access_boolean | |
| owner | MAIN DE-863 DE-BY-FWS |
| owner_facet | MAIN DE-863 DE-BY-FWS |
| physical | 1 online resource (vi, 264 pages) : illustrations |
| psigel | ZDB-4-EBA |
| publishDate | 2013 |
| publishDateSearch | 2013 |
| publishDateSort | 2013 |
| publisher | Greenleaf Publishing, |
| record_format | marc |
| spelling | Rare diseases : challenges and opportunities for social entrepreneurs / edited by Nicolas Sireau. Sheffield, England : Greenleaf Publishing, 2013. 1 online resource (vi, 264 pages) : illustrations text txt rdacontent computer c rdamedia online resource cr rdacarrier 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. Rare diseases. http://id.loc.gov/authorities/subjects/sh92005613 Pharmaceutical industry. Europe. http://id.loc.gov/authorities/subjects/sh85045631 Rare Diseases drug therapy https://id.nlm.nih.gov/mesh/D035583Q000188 Entrepreneurship organization & administration https://id.nlm.nih.gov/mesh/D016520Q000458 Orphan Drug Production methods https://id.nlm.nih.gov/mesh/D009965Q000379 Europe https://id.nlm.nih.gov/mesh/D005060 Drug Industry https://id.nlm.nih.gov/mesh/D004345 Maladies rares. Industrie pharmaceutique. Europe. HEALTH & FITNESS Diseases General. bisacsh MEDICAL Clinical Medicine. bisacsh MEDICAL Diseases. bisacsh MEDICAL Evidence-Based Medicine. bisacsh MEDICAL Internal Medicine. bisacsh Pharmaceutical industry fast Rare diseases fast Europe fast https://id.oclc.org/worldcat/entity/E39PBJxCxPbbk4CPJDQJb4r6rq Sireau, Nicolas, 1973- editor. https://id.oclc.org/worldcat/entity/E39PCjH3RDK8H98MhQkHwhjw83 http://id.loc.gov/authorities/names/n2008059273 has work: Rare diseases (Text) https://id.oclc.org/worldcat/entity/E39PCH9b6BbhKt4hBBXy4fWjvd https://id.oclc.org/worldcat/ontology/hasWork Print version: Rare diseases. Sheffield, UK : Greenleaf Publishing, [2013] 9781906093525 (OCoLC)827952034 FWS01 ZDB-4-EBA FWS_PDA_EBA https://search.ebscohost.com/login.aspx?direct=true&scope=site&db=nlebk&AN=760652 Volltext |
| spellingShingle | Rare diseases : challenges and opportunities for social entrepreneurs / 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. Rare diseases. http://id.loc.gov/authorities/subjects/sh92005613 Pharmaceutical industry. Rare Diseases drug therapy https://id.nlm.nih.gov/mesh/D035583Q000188 Entrepreneurship organization & administration https://id.nlm.nih.gov/mesh/D016520Q000458 Orphan Drug Production methods https://id.nlm.nih.gov/mesh/D009965Q000379 Drug Industry https://id.nlm.nih.gov/mesh/D004345 Maladies rares. Industrie pharmaceutique. HEALTH & FITNESS Diseases General. bisacsh MEDICAL Clinical Medicine. bisacsh MEDICAL Diseases. bisacsh MEDICAL Evidence-Based Medicine. bisacsh MEDICAL Internal Medicine. bisacsh Pharmaceutical industry fast Rare diseases fast |
| subject_GND | http://id.loc.gov/authorities/subjects/sh92005613 http://id.loc.gov/authorities/subjects/sh85045631 https://id.nlm.nih.gov/mesh/D035583Q000188 https://id.nlm.nih.gov/mesh/D016520Q000458 https://id.nlm.nih.gov/mesh/D009965Q000379 https://id.nlm.nih.gov/mesh/D005060 https://id.nlm.nih.gov/mesh/D004345 |
| title | Rare diseases : challenges and opportunities for social entrepreneurs / |
| title_auth | Rare diseases : challenges and opportunities for social entrepreneurs / |
| title_exact_search | Rare diseases : challenges and opportunities for social entrepreneurs / |
| title_full | Rare diseases : challenges and opportunities for social entrepreneurs / edited by Nicolas Sireau. |
| title_fullStr | Rare diseases : challenges and opportunities for social entrepreneurs / edited by Nicolas Sireau. |
| title_full_unstemmed | Rare diseases : challenges and opportunities for social entrepreneurs / edited by Nicolas Sireau. |
| title_short | Rare diseases : |
| title_sort | rare diseases challenges and opportunities for social entrepreneurs |
| title_sub | challenges and opportunities for social entrepreneurs / |
| topic | Rare diseases. http://id.loc.gov/authorities/subjects/sh92005613 Pharmaceutical industry. Rare Diseases drug therapy https://id.nlm.nih.gov/mesh/D035583Q000188 Entrepreneurship organization & administration https://id.nlm.nih.gov/mesh/D016520Q000458 Orphan Drug Production methods https://id.nlm.nih.gov/mesh/D009965Q000379 Drug Industry https://id.nlm.nih.gov/mesh/D004345 Maladies rares. Industrie pharmaceutique. HEALTH & FITNESS Diseases General. bisacsh MEDICAL Clinical Medicine. bisacsh MEDICAL Diseases. bisacsh MEDICAL Evidence-Based Medicine. bisacsh MEDICAL Internal Medicine. bisacsh Pharmaceutical industry fast Rare diseases fast |
| topic_facet | Rare diseases. Pharmaceutical industry. Europe. Rare Diseases drug therapy Entrepreneurship organization & administration Orphan Drug Production methods Europe Drug Industry Maladies rares. Industrie pharmaceutique. HEALTH & FITNESS Diseases General. MEDICAL Clinical Medicine. MEDICAL Diseases. MEDICAL Evidence-Based Medicine. MEDICAL Internal Medicine. Pharmaceutical industry Rare diseases |
| url | https://search.ebscohost.com/login.aspx?direct=true&scope=site&db=nlebk&AN=760652 |
| work_keys_str_mv | AT sireaunicolas rarediseaseschallengesandopportunitiesforsocialentrepreneurs |