000			05776cam a2200925Ia 4500
001			ocn862577543
003			OCoLC
005			20220711212527.0
006			m o d
007			cr |||||||||||
008			131109s2013 uika ob 000 0 eng d
040			_aDKDLA _beng _epn _cDKDLA _dOCLCO _dSFB _dN$T _dOCLCF _dORZ _dYDXCP _dEBLCP _dE7B _dDEBSZ _dCOO _dCUS _dOCLCQ _dICA _dUKGRL _dAGLDB _dMERUC _dOCLCQ _dYDX _dM8D _dU3W _dVTS _dOCLCA _dOCLCQ _dYOU _dSTF _dAU@ _dUKAHL _dOCLCQ _dOCLCO _dOCLCQ _dOCLCO
019			_a884015531 _a1260363594
020			_a9781909493209 _q(electronic bk.)
020			_a1909493201 _q(electronic bk.)
020			_a9781906093525 _q(paperback)
020			_a1906093520 _q(paperback)
029	1		_aAU@ _b000055219604
029	1		_aAU@ _b000058046232
029	1		_aAU@ _b000062621025
029	1		_aDEBBG _bBV042794757
029	1		_aDEBSZ _b410563463
029	1		_aDEBSZ _b481276890
029	1		_aDKDLA _b820120-katalog:000618784
029	1		_aNZ1 _b16047637
035			_a(OCoLC)862577543 _z(OCoLC)884015531 _z(OCoLC)1260363594
043			_ae------
050		4	_aRC48.8 _b.R37 2013
060		4	_a2013 F-056
060		4	_aQZ 140
072		7	_aHEA _x039000 _2bisacsh
072		7	_aMED _x014000 _2bisacsh
072		7	_aMED _x022000 _2bisacsh
072		7	_aMED _x112000 _2bisacsh
072		7	_aMED _x045000 _2bisacsh
082	0	4	_a616 _223
049			_aMAIN
245	0	0	_aRare diseases : _bchallenges and opportunities for social entrepreneurs / _cedited by Nicolas Sireau.
260			_aSheffield, England : _bGreenleaf Publishing, _c2013.
300			_a1 online resource (vi, 264 pages) : _billustrations
336			_atext _btxt _2rdacontent
337			_acomputer _bc _2rdamedia
338			_aonline resource _bcr _2rdacarrier
504			_aIncludes bibliographical references.
520	8		_aThere 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		_aPrint version record.
505	0		_aCover; 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		_a8 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.
590			_aeBooks on EBSCOhost _bEBSCO eBook Subscription Academic Collection - Worldwide
650		0	_aRare diseases. _9884992
650		0	_aPharmaceutical industry. _97667
651		0	_aEurope.
650	1	2	_aRare Diseases _xdrug therapy _9975197
650	2	2	_aEntrepreneurship _xorganization & administration _9975198
650	2	2	_aOrphan Drug Production _xmethods _9975199
651		2	_aEurope
650		2	_aDrug Industry _9939152
650		6	_aMaladies rares. _9975200
650		6	_aIndustrie pharmaceutique. _9939154
651		6	_aEurope.
650		7	_aHEALTH & FITNESS _xDiseases _xGeneral. _2bisacsh _988045
650		7	_aMEDICAL _xClinical Medicine. _2bisacsh _991637
650		7	_aMEDICAL _xDiseases. _2bisacsh _988037
650		7	_aMEDICAL _xEvidence-Based Medicine. _2bisacsh _991638
650		7	_aMEDICAL _xInternal Medicine. _2bisacsh _991639
650		7	_aPharmaceutical industry. _2fast _0(OCoLC)fst01060129 _97667
650		7	_aRare diseases. _2fast _0(OCoLC)fst01090123 _9884992
655		4	_aElectronic books.
700	1		_aSireau, Nicolas, _d1973- _eeditor. _9389531
776	0	8	_iPrint version: _tRare diseases. _dSheffield, UK : Greenleaf Publishing, [2013] _z9781906093525 _w(OCoLC)827952034
856	4	0	_uhttps://search.ebscohost.com/login.aspx?direct=true&scope=site&db=nlebk&AN=760652
938			_aAskews and Holts Library Services _bASKH _nAH33322407
938			_aEBL - Ebook Library _bEBLB _nEBL1741715
938			_aebrary _bEBRY _nebr10890740
938			_aEBSCOhost _bEBSC _n760652
938			_aGreenleaf Publishing _bUKGR _n9781909493209
938			_aYBP Library Services _bYANK _n14975909
938			_aYBP Library Services _bYANK _n11789726
994			_a92 _bINOPJ
999			_c2761662 _d2761662