Drug Discovery - Orphan Drugs and Rare Diseases
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21

(FIVE YEARS 0)

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2

(FIVE YEARS 0)

Published By Royal Society Of Chemistry

9781849738064

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Chapter 12. Unleashing the Power of Semi-Synthesis: The Discovery of Torisel®

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00347 ◽

2017 ◽

pp. 347-366

Author(s):

Jerauld S. Skotnicki ◽

Magid A. Abou-Gharbia

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Chapter 11. Drug Discovery Approaches for Rare Neuromuscular Diseases

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00257 ◽

2014 ◽

pp. 257-343 ◽

Author(s):

Graham M. Wynne ◽

Angela J. Russell

Keyword(s):

Drug Discovery ◽

Neuromuscular Diseases

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Chapter 9. Discovery and Development of Tafamidis for the Treatment of TTR Familial Amyloid Polyneuropathy

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00202 ◽

2014 ◽

pp. 202-229

Author(s):

Richard Labaudinière

Keyword(s):

Familial Amyloid Polyneuropathy ◽

Amyloid Polyneuropathy

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Chapter 7. Discovery and Clinical Development of Idursulfase (Elaprase®) for the Treatment of Mucopolysaccharidosis II (Hunter Syndrome)

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00164 ◽

2014 ◽

pp. 164-182 ◽

Author(s):

Michael Heartlein ◽

Alan Kimura

Keyword(s):

Hunter Syndrome ◽

Clinical Development ◽

Mucopolysaccharidosis Ii

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Front Matter

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-fp001 ◽

2014 ◽

pp. P001-P006

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Subject Index

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00448 ◽

2014 ◽

pp. 448-463

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Chapter 5. Disease Advocacy Organisations

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00115 ◽

2014 ◽

pp. 115-138

Author(s):

Sharon F. Terry ◽

Caroline Kant

Keyword(s):

Disease Advocacy

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Chapter 3. The Challenges of Conducting Clinical Trials in Diseases with Small Target Populations

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00053 ◽

2014 ◽

pp. 53-79

Author(s):

Steven Arkin

Keyword(s):

Clinical Trials ◽

Small Target ◽

Target Populations

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Chapter 13. Treatments for Pulmonary Arterial Hypertension

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00369 ◽

2014 ◽

pp. 369-397

Author(s):

Michael J. Palmer

Keyword(s):

Pulmonary Arterial Hypertension ◽

Arterial Hypertension ◽

Pulmonary Arterial

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Chapter 4. Treating Rare Diseases: Business Model for Orphan Drug Development

Drug Discovery - Orphan Drugs and Rare Diseases ◽

10.1039/9781782624202-00083 ◽

2014 ◽

pp. 83-111

Author(s):

Cory Williams

Keyword(s):

Drug Development ◽

Business Model ◽

Orphan Drug ◽

Rare Diseases ◽

Orphan Drug Development

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