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Volume 5, Issue 1

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 5, Issue 1 (2017)

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Expert Opinion on Orphan Drugs, Volume 5, Issue 1 (2017)

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Editorial

Therapeutic targets in homocystinuria due to cystathionine β-synthase deficiency: new European guidelines

Bridget Wilcken

Pages: 1-3

Published online: 21 Nov 2016

First Page PreviewforTherapeutic targets in homocystinuria due to cystathionine β-synthase deficiency: new European guidelines | Full Text | References | PDF (293 KB)

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Review

Genetic therapy of Xeroderma Pigmentosum: analysis of strategies and translation

Maria Goncalves-Maia & Thierry Magnaldo

Pages: 5-17

Published online: 18 Nov 2016

AbstractforGenetic therapy of Xeroderma Pigmentosum: analysis of strategies and translation | Full Text | References | PDF (1.1 MB)

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Review

Managing post-transplant lymphoproliferative disorder

Jan Styczynski

Pages: 19-35

Published online: 24 Nov 2016

AbstractforManaging post-transplant lymphoproliferative disorder | Full Text | References | PDF (1.5 MB)

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Review

Epidemiological research, scientific research, and applied research on rare diseases in China: current challenges and prospects for the future

Qi Tang, Peipei Song, Lin Mei, Lingzhong Xu & Wei Tang

Pages: 37-44

Published online: 18 Nov 2016

AbstractforEpidemiological research, scientific research, and applied research on rare diseases in China: current challenges and prospects for the future | Full Text | References | PDF (840.5 KB)

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Review

Pathophysiology, current treatments and future targets in hereditary forms of renal Fanconi syndrome

Enriko Klootwijk, Stephanie Dufek, Naomi Issler, Detlef Bockenhauer & Robert Kleta

Pages: 45-54

Published online: 30 Nov 2016

AbstractforPathophysiology, current treatments and future targets in hereditary forms of renal Fanconi syndrome | Full Text | References | PDF (1.1 MB)

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Review

Immunotherapies in phase II and III trials for the treatment of systemic lupus erythematosus

Massimo Radin, Irene Cecchi, Karen Schreiber, Simone Baldovino, Daniela Rossi, Elisa Menegatti, Dario Roccatello & Savino Sciascia

Pages: 55-69

Published online: 18 Nov 2016

AbstractforImmunotherapies in phase II and III trials for the treatment of systemic lupus erythematosus | Full Text | References | PDF (1.2 MB)

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Review

Rare tumors in gynaecological cancers and the lack of therapeutic options and clinical trials

Victoria Mandilaras, Katherine Karakasis, Blaise Clarke, Amit Oza & Stephanie Lheureux

Pages: 71-83

Published online: 05 Dec 2016

AbstractforRare tumors in gynaecological cancers and the lack of therapeutic options and clinical trials | Full Text | References | PDF (962.6 KB)

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Drug Evaluation

Article

Immunotherapy for malignant primary brain tumors with ICT-107, a dendritic cell vaccine

Alia Hdeib & Andrew Sloan

Pages: 85-89

Published online: 10 Dec 2016

AbstractforImmunotherapy for malignant primary brain tumors with ICT-107, a dendritic cell vaccine | Full Text | References | PDF (432.4 KB)

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Article

Imetelstat for treatment of myelofibrosis

E. Virtgaym, D. Tremblay, C. Iancu-Rubin, R. Hoffman & J. Mascarenhas

Pages: 91-97

Published online: 10 Dec 2016

AbstractforImetelstat for treatment of myelofibrosis | Full Text | References | PDF (670.9 KB)

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Article

Rituximab in the treatment of pemphigus

Shawn Shetty & A. Razzaque Ahmed

Pages: 99-109

Published online: 27 Dec 2016

AbstractforRituximab in the treatment of pemphigus | Full Text | References | PDF (911.9 KB)

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Corrigendum

Correction

Corrigendum

Page: i

Published online: 18 Aug 2016

CitationforCorrigendum | Full Text | PDF (87 KB)

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