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Expert Opinion on Orphan Drugs

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

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

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Editorial

Treatment of Wilson’s disease – another point of view

Anna Członkowska MD PhD & Tomasz Litwin

Pages: 239-243

Published online: 20 Feb 2015

First Page PreviewforTreatment of Wilson’s disease – another point of view | Full Text | References | PDF (148.5 KB)

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Review

Next-generation treatment of acquired hemophilia A

Massimo Franchini MD

Pages: 245-251

Published online: 19 Feb 2015

AbstractforNext-generation treatment of acquired hemophilia A | Full Text | References | PDF (181.4 KB)

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Pathophysiology, genetics, clinical features, diagnosis and therapeutic trials in progressive supranuclear palsy

Brent Bluett & Irene Litvan

Pages: 253-265

Published online: 20 Feb 2015

AbstractforPathophysiology, genetics, clinical features, diagnosis and therapeutic trials in progressive supranuclear palsy | Full Text | References | PDF (365.6 KB)

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Pathogenesis, epidemiology, diagnosis and clinical aspects of Smith–Lemli–Opitz syndrome

Simona E Bianconi MD, Joanna L Cross, Christopher A Wassif & Forbes D Porter MD PhD

Pages: 267-280

Published online: 16 Feb 2015

AbstractforPathogenesis, epidemiology, diagnosis and clinical aspects of Smith–Lemli–Opitz syndrome | Full Text | References | PDF (713.2 KB) | Supplemental

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Pathogenesis and management of type 2 neurofibromatosis

Martin G McCabe PhD & Dafydd Gareth Evans MD

Pages: 281-292

Published online: 15 Feb 2015

AbstractforPathogenesis and management of type 2 neurofibromatosis | Full Text | References | PDF (276.8 KB)

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Enzyme replacement therapy: lessons learned and emerging questions

Agnieszka Jurecka MD PhD & Anna Tylki-Szymańska MD PhD

Pages: 293-305

Published online: 18 Feb 2015

AbstractforEnzyme replacement therapy: lessons learned and emerging questions | Full Text | References | PDF (314.3 KB)

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Diagnosis, classification and treatment of mucopolysaccharidosis type I

Eveline J Langereis MD, Naomi van Vlies MSc & Frits A Wijburg (Professor) MD

Pages: 307-320

Published online: 20 Feb 2015

AbstractforDiagnosis, classification and treatment of mucopolysaccharidosis type I | Full Text | References | PDF (433.3 KB)

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Orphan Drug Designation

Fresh from the designation pipeline: orphan drugs recently designated in the European Union (August–October 2014)

Sabina Antoniu (Lecturer) MD, PhD

Pages: 321-328

Published online: 19 Feb 2015

AbstractforFresh from the designation pipeline: orphan drugs recently designated in the European Union (August–October 2014) | Full Text | References | PDF (175.1 KB)

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Drug Evaluation

A closer look at nerve growth factor: from biology to clinical trials in ophthalmology

Flavio Mantelli MD PhD, Marta Sacchetti MD PhD, Gianluca Scuderi MD & Alessandro Lambiase (Professor) MD PhD

Pages: 329-341

Published online: 21 Jan 2015

AbstractforA closer look at nerve growth factor: from biology to clinical trials in ophthalmology | Full Text | References | PDF (270.8 KB)

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Tivantinib for hepatocellular carcinoma

Lorenza Rimassa MD, Nicola Personeni MD & Armando Santoro MD

Pages: 343-351

Published online: 13 Feb 2015

AbstractforTivantinib for hepatocellular carcinoma | Full Text | References | PDF (337 KB)

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

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

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

Editorial

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Orphan Drug Designation

Drug Evaluation

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