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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 1, Issue 1 (2013)

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Expert Opinion on Orphan Drugs, Volume 1, Issue 1 (2013)

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Editorial

Editorial

Big Pharma's new model in orphan drugs and rare diseases

M Ian Phillips (Norris Professor of Life Sciences, Director) PhD, DSc, FAHA

Pages: 1-3

Published online: 17 Dec 2012

AbstractforBig Pharma's new model in orphan drugs and rare diseases | Full Text | References | PDF (158.6 KB)

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Reviews

Review Article

Emerging drug targets in amyotrophic lateral sclerosis

Michael P Bova (Senior Director of Target Advancement) PhD & Gene G Kinney (Senior Vice President) PhD

Pages: 5-20

Published online: 17 Dec 2012

AbstractforEmerging drug targets in amyotrophic lateral sclerosis | Full Text | References | PDF (332.6 KB)

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Review Article

Orphan drugs for the treatment of hairy cell leukemia

Estella Matutes MD PhD FRCPath

Pages: 21-31

Published online: 17 Dec 2012

AbstractforOrphan drugs for the treatment of hairy cell leukemia | Full Text | References | PDF (460.8 KB)

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Review Article

Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy

Michele A Scully MD, Shree Pandya MS PT & Richard T Moxley MD

Pages: 33-46

Published online: 17 Dec 2012

AbstractforReview of Phase II and Phase III clinical trials for Duchenne muscular dystrophy | Full Text | References | PDF (229.3 KB)

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Review Article

Phase II and III drugs for the treatment of fragile X syndrome

Laura C Politte & Christopher J McDougle

Pages: 47-65

Published online: 17 Dec 2012

AbstractforPhase II and III drugs for the treatment of fragile X syndrome | Full Text | References | PDF (413.9 KB)

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Review Article

Understanding molecular pathways and the identification of subgroups of patients with specific biomarker predictors of drug response related to orphan drugs

Ciara Kelly (Medical Oncology Specialist Registrar) MB BCh BAO (Hons) MRCPI & Catherine M Kelly (Consultant Medical Oncologist) (UCD Clinical Lecturer) MB BCh BAO BSc (Hons) MRCPI MSc (Clin. Epi)

Pages: 67-76

Published online: 17 Dec 2012

AbstractforUnderstanding molecular pathways and the identification of subgroups of patients with specific biomarker predictors of drug response related to orphan drugs | Full Text | References | PDF (398.5 KB)

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Drug Evaluations

Article

The erythropoietin analog ARA 290 for treatment of sarcoidosis-induced chronic neuropathic pain

Marieke Niesters, Maarten Swartjes, Lara Heij, Michael Brines, Anthony Cerami, Ann Dunne, Elske Hoitsma & Albert Dahan

Pages: 77-87

Published online: 17 Dec 2012

AbstractforThe erythropoietin analog ARA 290 for treatment of sarcoidosis-induced chronic neuropathic pain | Full Text | References | PDF (566.7 KB)

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Article

Evaluation of idursulfase for the treatment of mucopolysaccharidosis II (Hunter syndrome)

Maurizio Scarpa MD PhD

Pages: 89-98

Published online: 17 Dec 2012

AbstractforEvaluation of idursulfase for the treatment of mucopolysaccharidosis II (Hunter syndrome) | Full Text | References | PDF (502.7 KB)

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Research Article

Article

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Page: i

Published online: 17 Dec 2012

CitationforRelated articles | Full Text | PDF (129.9 KB)

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