Rare Diseases
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Published By Greenleaf Publishing Limited

9781909493209

Latest Documents Most Cited Documents Contributed Authors Related Sources Related Keywords

Drug repositioning strategies for rare and orphan diseases: A cost-effective approach for new uses for existing drugs

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_8 ◽

2014 ◽

pp. 114-149

Author(s):

Maria P. del Castillo-Frias ◽

Andrew J. Doig ◽

Farid Khan

Keyword(s):

Drug Repositioning ◽

Cost Effective ◽

Orphan Diseases ◽

Cost Effective Approach

The practicalities of clinical development of drugs for rare diseases

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_6 ◽

2014 ◽

pp. 62-86

Author(s):

Anthony K. Hall

Keyword(s):

Rare Diseases ◽

Clinical Development

The challenges of scaling up an orphan drug enterprise

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_4 ◽

2014 ◽

pp. 22-42

Author(s):

Hans Schikan

Keyword(s):

Orphan Drug ◽

Scaling Up

Supplementary Material

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_16 ◽

2014 ◽

pp. 263-272

Author(s):

Nicolas Sireau

Keyword(s):

Supplementary Material

Lobbying for a national rare disease plan in the UK: Lessons for rare disease patient alliances

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_14 ◽

2014 ◽

pp. 207-230

Author(s):

Stephen Nutt

Keyword(s):

Rare Disease ◽

Disease Patient ◽

The Uk ◽

Rare Disease Patient

Introduction

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_2 ◽

2014 ◽

pp. 1-6

Author(s):

Nicolas Sireau

Preliminary Material

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_1 ◽

2014 ◽

pp. i-vi

Author(s):

Nicolas Sireau

Toward a focused, multinational, rare disease awareness initiative

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_3 ◽

2014 ◽

pp. 7-21

Author(s):

Peter Saltonstall ◽

E. Michael D. Scott

Keyword(s):

Rare Disease ◽

Disease Awareness

The rise and fall of Sanfilippo syndrome

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_13 ◽

2014 ◽

pp. 201-206

Author(s):

Karen Aiach

Keyword(s):

Sanfilippo Syndrome

The BLACKSWAN Foundation for rare diseases

Rare Diseases ◽

10.9774/gleaf.978-1-909493-20-9_12 ◽

2014 ◽

pp. 193-200

Author(s):

Olivier Menzel ◽

Silvia Panigone

Keyword(s):

Rare Diseases