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Volume 3, Issue 10

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 10 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 10 (2015)

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Editorial

Complexities in transitioning a child with a rare disorder

José T Morales

Pages: 1097-1100

Published online: 18 Aug 2015

AbstractforComplexities in transitioning a child with a rare disorder | Full Text | References | PDF (132.7 KB)

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Free Access

Review

The challenging treatment of Behcet’s disease

Ina Kötter (Professor) MD

Pages: 1101-1110

Published online: 24 Sep 2015

AbstractforThe challenging treatment of Behcet’s disease | Full Text | References | PDF (267.3 KB)

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Considering chemotherapy in synovial sarcoma

Andrea Ferrari MD, Stefano Chiaravalli MD, Michela Casanova MD, Patrizia Gasparini PhD, Nadège Corradini MD & Daniel Orbach MD

Pages: 1111-1124

Published online: 06 Aug 2015

AbstractforConsidering chemotherapy in synovial sarcoma | Full Text | References | PDF (509.3 KB)

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Cord blood transplantation in children with hemoglobinopathies

Luisa Strocchio, Mariateresa Romano, Maria Giuseppina Cefalo, Luciana Vinti, Stefania Gaspari & Franco Locatelli MD PhD

Pages: 1125-1136

Published online: 10 Aug 2015

AbstractforCord blood transplantation in children with hemoglobinopathies | Full Text | References | PDF (266.5 KB)

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New therapeutic targets in rare genetic skeletal diseases

Michael D Briggs (Professor of Skeletal Genetics) PhD, Peter A Bell PhD, Michael J Wright MB ChB MSc FRCP & Katarzyna A Pirog PhD

Pages: 1137-1154

Published online: 24 Sep 2015

AbstractforNew therapeutic targets in rare genetic skeletal diseases | Full Text | References | PDF (659.7 KB)

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Open Access

Current and emerging therapy for improving outcomes in patients with intraocular retinoblastoma

Rachel C Brennan (Assistant Member) , Eleanor M Pritchard, R Kiplin Guy, Michael A Dyer & Matthew W Wilson

Pages: 1155-1166

Published online: 06 Aug 2015

AbstractforCurrent and emerging therapy for improving outcomes in patients with intraocular retinoblastoma | Full Text | References | PDF (687.2 KB)

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Prospective therapeutic approaches in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)

Raquel Cabrera-Pérez BSc, Javier Torres-Torronteras PhD, Ferran Vila-Julià BSc, Francisco J Ortega PhD, Yolanda Cámara PhD, Jordi Barquinero MD PhD & Ramon Martí PhD

Pages: 1167-1182

Published online: 16 Sep 2015

AbstractforProspective therapeutic approaches in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) | Full Text | References | PDF (896.4 KB)

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Prospects of gene and cell therapy for managing cardiac complications in Friedreich ataxia

Charles J Isaacs, Julianna E Shinnick, Kimberly Schadt, David R Lynch & Kimberly Y Lin

Pages: 1183-1196

Published online: 31 Aug 2015

AbstractforProspects of gene and cell therapy for managing cardiac complications in Friedreich ataxia | Full Text | References | PDF (699 KB)

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Drug Evaluation

Retraction

RETRACTED ARTICLE: 5-Azacitidine for treating acute myelogenous leukemia

Riad O El Fakih MD, Richard Champlin & Betul Oran

Pages: 1197-1207

Published online: 24 Sep 2015

First Page PreviewforRETRACTED ARTICLE: 5-Azacitidine for treating acute myelogenous leukemia | PDF (355.1 KB)

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Free Access

Crizotinib: an orphan drug for treating non-small-cell lung cancer

Siddhartha Devarakonda, Bharath Ganesh, Janelle Mann & Ramaswamy Govindan

Pages: 1209-1218

Published online: 24 Sep 2015

AbstractforCrizotinib: an orphan drug for treating non-small-cell lung cancer | Full Text | References | PDF (221.5 KB)

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Denosumab for treating giant cell tumor of bone

Ewelina Jagiello-Wieczorek, Andrzej Pieńkowski & Piotr Rutkowski MD PhD

Pages: 1219-1229

Published online: 10 Sep 2015

AbstractforDenosumab for treating giant cell tumor of bone | Full Text | References | PDF (662.7 KB)

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Use of romidepsin for the treatment of mycosis fungoides and Sezary syndrome – role of romidepsin in the current therapeutic landscape and implications for future practice

Carrie van der Weyden, Michael Dickinson, Susan E Bates & Henry Miles Prince

Pages: 1231-1239

Published online: 16 Sep 2015

AbstractforUse of romidepsin for the treatment of mycosis fungoides and Sezary syndrome – role of romidepsin in the current therapeutic landscape and implications for future practice | Full Text | References | PDF (433.2 KB)

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Expert Opinion on Orphan Drugs (Vol.3, Iss.10, 2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 10 (2015)

Expert Opinion on Orphan Drugs, Volume 3, Issue 10 (2015)

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