Expert Opinion on Orphan Drugs, Volume 1, Issue 6 (2013)

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Expert Opinion on Orphan Drugs, Volume 1, Issue 6 (2013)

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Original Research

Article
Changes in disease activity, lung function and quality of life in patients with refractory sarcoidosis after anti-TNF treatment
Henricus NAJ van Rijswijk MD, Adriane DM Vorselaars MD, Henk JT Ruven PhD, Ruth GM Keijsers MD PhD, Pieter Zanen MD PhD, Ingrid HE Korenromp PhD & Jan C Grutters MD PhD
Pages: 437-443
Published online: 07 May 2013
AbstractforChanges in disease activity, lung function and quality of life in patients with refractory sarcoidosis after anti-TNF treatment | Full Text | References | PDF (380.8 KB)
  • 119 Views
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Reviews

Review Article
Treatment of dry eyes in Sjögren's syndrome: the role of autologous blood serum
Ioannis Katsakoulas BSc, Clio P Mavragani MD & Haralampos M Moutsopoulos MD FRCP FACP
Pages: 445-456
Published online: 04 May 2013
AbstractforTreatment of dry eyes in Sjögren's syndrome: the role of autologous blood serum | Full Text | References | PDF (279.5 KB)
  • 68 Views
  • 1 CrossRef citations
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Review Article
Pharmacotherapy of Pompe disease
Giancarlo Parenti (Associate Professor) MD & Generoso Andria (Professor of Pediatrics, Head of the Department of Clinical Pediatrics) MD
Pages: 457-471
Published online: 06 May 2013
AbstractforPharmacotherapy of Pompe disease | Full Text | References | PDF (603 KB)
  • 127 Views
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Review Article
Repurposing as a strategy for orphan drug development, evidence from European approvals
Peter Norman MBA PhD
Pages: 473-480
Published online: 04 May 2013
AbstractforRepurposing as a strategy for orphan drug development, evidence from European approvals | Full Text | References | PDF (376.7 KB)
  • 250 Views
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Review Article
Pharmacotherapy of von Willebrand disease
Massimo Franchini MD & Giuseppe Lippi
Pages: 481-489
Published online: 11 May 2013
AbstractforPharmacotherapy of von Willebrand disease | Full Text | References | PDF (394.2 KB)
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Drug Evaluation

Nitisinone for the treatment of hereditary tyrosinemia type I
Patrick James McKiernan (Consultant Pediatrician) BSc MB BCh MRCP(UK) FRCPCH
Pages: 491-497
Published online: 21 May 2013
Abstractfor Nitisinone for the treatment of hereditary tyrosinemia type I | Full Text | References | PDF (256 KB)
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Orphan Drug Designations

Article
Fresh from the designation pipeline: orphan drugs recently designated in the European Union (November 2012 – January 2013)
Sabina Antoniu (Lecturer) MD PhD
Pages: 499-505
Published online: 08 May 2013
AbstractforFresh from the designation pipeline: orphan drugs recently designated in the European Union (November 2012 – January 2013) | Full Text | References | PDF (232.3 KB)
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Research Article

Article
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Page: i
Published online: 21 May 2013
CitationforRelated articles | Full Text | PDF (169.7 KB)
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