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Volume 1, Issue 9

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 1, Issue 9 (2013)

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Expert Opinion on Orphan Drugs, Volume 1, Issue 9 (2013)

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Editorials

Editorial

Is orphan drug pricing blowing a bubble? The unique situation of orphan drugs and why high prices will likely persist

John-Paul Michaud, Robin Modi & M Ian Phillips

Pages: 675-679

Published online: 21 Aug 2013

AbstractforIs orphan drug pricing blowing a bubble? The unique situation of orphan drugs and why high prices will likely persist | Full Text | References | PDF (231.9 KB)

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Editorial

Rare diseases and orphan drugs in Japan: developing multiple strategies of regulation and research

Peipei Song, Wei Tang & Norihiro Kokudo

Pages: 681-683

Published online: 26 Aug 2013

AbstractforRare diseases and orphan drugs in Japan: developing multiple strategies of regulation and research | Full Text | References | PDF (216.5 KB)

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Review

Review Article

Management options for Takayasu arteritis

Enrico Tombetti, Angelo Manfredi, Maria Grazia Sabbadini & Elena Baldissera

Pages: 685-693

Published online: 21 Aug 2013

AbstractforManagement options for Takayasu arteritis | Full Text | References | PDF (341.2 KB)

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Reviews

Review Article

Management of CADASIL syndrome

Michele Ragno MD & Luigi Trojano MD

Pages: 695-703

Published online: 13 Aug 2013

AbstractforManagement of CADASIL syndrome | Full Text | References | PDF (346 KB)

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Review Article

Gene therapy for Wiskott–Aldrich syndrome

Maximilian Georg Wolfgang Witzel, Christian Joerg Braun, Kaan Boztug & Christoph Klein

Pages: 705-715

Published online: 13 Aug 2013

AbstractforGene therapy for Wiskott–Aldrich syndrome | Full Text | References | PDF (669.5 KB)

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Review Article

Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome)

Olga LM Meijer MD PhD, Naomi van Vlies PhD & Frits A Wijburg

Pages: 717-730

Published online: 13 Aug 2013

AbstractforTreatment of mucopolysaccharidosis type III (Sanfilippo syndrome) | Full Text | References | PDF (520.8 KB)

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Review Article

Treatment options for graft-versus-host disease

Inken Hilgendorf & Daniel Wolff

Pages: 731-743

Published online: 21 Aug 2013

AbstractforTreatment options for graft-versus-host disease | Full Text | References | PDF (415.5 KB)

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Drug Evaluations

Product Review

Omacetaxine mepesuccinate for management of chronic myeloid leukemia

Jeffrey H Lipton MD PhD & Dennis (Dong Hwan) Kim MD PhD

Pages: 745-754

Published online: 13 Aug 2013

Abstractfor Omacetaxine mepesuccinate for management of chronic myeloid leukemia | Full Text | References | PDF (417.4 KB)

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Product Review

Use of gevokizumab for the treatment of Behçet's disease

Nicholas A Barnes PhD, Maya H Buch MBChB PhD FRCP & Michael F McDermott MBChB MRCPI DMed

Pages: 755-763

Published online: 28 Aug 2013

AbstractforUse of gevokizumab for the treatment of Behçet's disease | Full Text | References | PDF (489.5 KB)

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Orphan Drug Designations

Product Review

Fresh from the designation pipeline: orphan drugs recently designated in the European Union (March – April 2013)

Sabina Antoniu (Lecturer) MD PhD

Pages: 765-768

Published online: 13 Aug 2013

AbstractforFresh from the designation pipeline: orphan drugs recently designated in the European Union (March – April 2013) | Full Text | References | PDF (217.9 KB)

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Original Articles

Article

Page: i

Published online: 28 Aug 2013

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