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Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 3, Issue 7 (2015)

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Expert Opinion on Orphan Drugs, Volume 3, Issue 7 (2015)

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Original Research

Article

Recruitment procedures for descriptive socio-economic studies in rare diseases. The BURQOL-RD project

Pedro Serrano-Aguilar, Renata Linertová, Manuel Posada-de-la-Paz, Julio López-Bastida, Nerea González-Hernández, Domenica Taruscio & BURQOL-RD Research Network*

Pages: 759-765

Published online: 17 Jun 2015

AbstractforRecruitment procedures for descriptive socio-economic studies in rare diseases. The BURQOL-RD project | Full Text | References | PDF (179.7 KB) | Supplemental

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Review

A pragmatic approach to imaging in large vessel vasculitis

Pravin Patil MBBS MRCP, Christian Dejaco MD PhD & Bhaskar Dasgupta MD FRCP

Pages: 767-775

Published online: 23 Jun 2015

AbstractforA pragmatic approach to imaging in large vessel vasculitis | Full Text | References | PDF (657 KB)

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Current and future treatment options for cutaneous lupus erythematosus

Christopher B Hansen (Assistant Professor) MD & Jeffrey P Callen (Professor of Medicine (Dermatology), Chief)

Pages: 777-786

Published online: 23 Jun 2015

AbstractforCurrent and future treatment options for cutaneous lupus erythematosus | Full Text | References | PDF (244.5 KB)

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Pathogenic mechanisms and the prospect of gene therapy for choroideremia

Ioannis S Dimopoulos, Stephanie Chan, Robert E MacLaren & Ian M MacDonald

Pages: 787-798

Published online: 10 May 2015

AbstractforPathogenic mechanisms and the prospect of gene therapy for choroideremia | Full Text | References | PDF (589.6 KB)

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‘Approved for use in uveitis’: drug approval for an orphan disease

Jamie Lynne Metzinger MS MPH & C Stephen Foster MD FACS FACR

Pages: 799-807

Published online: 12 Jun 2015

Abstractfor‘Approved for use in uveitis’: drug approval for an orphan disease | Full Text | References | PDF (188.9 KB)

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Guillain-Barré syndrome: advances in pathogenic understanding and diagnostic improvements

Anna Hiu Yi Wong MRCP & Nobuhiro Yuki (Professor) MD PhD

Pages: 809-819

Published online: 15 May 2015

AbstractforGuillain-Barré syndrome: advances in pathogenic understanding and diagnostic improvements | Full Text | References | PDF (248.3 KB)

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Investigational treatments and therapeutic targets in Becker muscular dystrophy

Masafumi Matsuo MD PhD

Pages: 821-829

Published online: 23 Jun 2015

AbstractforInvestigational treatments and therapeutic targets in Becker muscular dystrophy | Full Text | References | PDF (452.6 KB)

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Hormone therapy in Fanconi anemia

Susan R Rose (Professor) MD

Pages: 831-842

Published online: 17 Jun 2015

AbstractforHormone therapy in Fanconi anemia | Full Text | References | PDF (231.5 KB)

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Drug Evaluation

Obinutuzumab: its use in the management of chronic lymphocytic leukemia

Jon E Arnason & Jennifer R Brown (Director)

Pages: 843-853

Published online: 26 May 2015

AbstractforObinutuzumab: its use in the management of chronic lymphocytic leukemia | Full Text | References | PDF (206.8 KB)

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Expert Opinion on Orphan Drugs (Vol.3, Iss.7, 2015)

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