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Volume 2, Issue 4

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 4 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 4 (2014)

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Editorial

A better fit? Biotech versus Big Pharma in orphan/rare disease drug research

Deborah A O’Neil (Chief Executive & Scientific Officer) PhD

Pages: 317-319

Published online: 22 Mar 2014

AbstractforA better fit? Biotech versus Big Pharma in orphan/rare disease drug research | Full Text | References | PDF (116 KB)

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Reviews

Review Article

Management of adult mastocytosis

Iván Alvarez-Twose, Almudena Matito, Laura Sánchez-Muñoz, José Mário Morgado & Luis Escribano

Pages: 321-336

Published online: 04 Feb 2014

AbstractforManagement of adult mastocytosis | Full Text | References | PDF (402.2 KB)

69 Views
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Review Article

Current status and perspectives in the treatment of Prader-Willi syndrome

Corin Badiu MD PhD & Otilia Mărginean MD PhD

Pages: 337-347

Published online: 06 Feb 2014

AbstractforCurrent status and perspectives in the treatment of Prader-Willi syndrome | Full Text | References | PDF (254.4 KB)

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Review Article

Management of acute intermittent porphyria

Pauline Harper MD PhD & Eliane Sardh MD PhD

Pages: 349-368

Published online: 28 Feb 2014

AbstractforManagement of acute intermittent porphyria | Full Text | References | PDF (483.3 KB)

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Review Article

Current and future therapeutic strategies for treatment of blastomycosis

Tyler Liebenstein PharmD & Jeannina A Smith MD

Pages: 369-379

Published online: 22 Feb 2014

AbstractforCurrent and future therapeutic strategies for treatment of blastomycosis | Full Text | References | PDF (259.8 KB)

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Review Article

Current and future therapeutic strategies for treating mixed cryoglobulinemia

David Saadoun MD PhD & Patrice Cacoub

Pages: 381-390

Published online: 13 Feb 2014

AbstractforCurrent and future therapeutic strategies for treating mixed cryoglobulinemia | Full Text | References | PDF (297 KB)

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Review Article

Orphan drugs for myelofibrosis

Claire N Harrison (Clinical Director Haematology and Oncology) & Michael Bennett (Director of Haematology)

Pages: 391-405

Published online: 13 Feb 2014

AbstractforOrphan drugs for myelofibrosis | Full Text | References | PDF (362.2 KB)

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Drug Evaluations

Evaluation of galsulfase for the treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Charles Marques Lourenço MD PhD & Roberto Giugliani (Professor) MD PhD

Pages: 407-417

Published online: 05 Mar 2014

AbstractforEvaluation of galsulfase for the treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) | Full Text | References | PDF (354.2 KB)

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Turoctocog alfa and drug development for hemophilia A

Steven R Lentz MD PhD, Stephanie Seremetis MD, Janice Staber MD & Roshni Kulkarni MD

Pages: 419-431

Published online: 01 Mar 2014

Abstractfor Turoctocog alfa and drug development for hemophilia A | Full Text | References | PDF (563.2 KB)

277 Views
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Research Article

Article

Page: i

Published online: 22 Mar 2014

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Expert Opinion on Orphan Drugs (Vol.2, Iss.4, 2014)

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