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Volume 2, Issue 8

Expert Opinion on Orphan Drugs

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Expert Opinion on Orphan Drugs, Volume 2, Issue 8 (2014)

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Expert Opinion on Orphan Drugs, Volume 2, Issue 8 (2014)

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Research Article

Article

Page: 1

Published online: 23 Jul 2014

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Editorials

Editorial

Recent orphan drugs that are first-in-class

M Ian Phillips & Jongserk R Lee

Pages: 759-763

Published online: 26 Jun 2014

AbstractforRecent orphan drugs that are first-in-class | Full Text | References | PDF (167.5 KB)

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3 CrossRef citations
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Free Access

Editorial

Coping with small populations of patients in clinical trials

Daniel J O’Connor (Expert Medical Assessor and UK Representative on COMP) BSc MB ChB MSc PhD MFPM & Robert James Hemmings (Unit Manager) (CHMP Member and Chair of SAWP) BSc MSc CStat

Pages: 765-768

Published online: 05 Jul 2014

AbstractforCoping with small populations of patients in clinical trials | Full Text | References | PDF (131.9 KB)

951 Views
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Reviews

Review Article

Treatment of idiopathic retroperitoneal fibrosis

Alessandra Palmisano MD PhD, Maria L Urban MD, Carlo Buzio MD & Augusto Vaglio MD PhD

Pages: 769-777

Published online: 06 Jun 2014

AbstractforTreatment of idiopathic retroperitoneal fibrosis | Full Text | References | PDF (278.8 KB)

42 Views
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Review Article

An update on congenital hyperinsulinism: advances in diagnosis and management

Jean-Baptiste Arnoux, Cécile Saint-Martin, Françoise Montravers, Virginie Verkarre, Louise Galmiche, Caroline Télion, Carmen Capito, Jean-Jacques Robert, Khalid Hussain, Yves Aigrain, Christine Bellanné-Chantelot & Pascale de Lonlay

Pages: 779-795

Published online: 31 May 2014

AbstractforAn update on congenital hyperinsulinism: advances in diagnosis and management | Full Text | References | PDF (859.1 KB)

110 Views
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Review Article

Rare disease landscape: will the blockbuster model be replaced?

Jonathan Stephens (Senior Oncology Drug Analyst) MPhil & Christine Blazynski (Chief Science Officer and Sr. Vice-President New Product Development) PhD

Pages: 797-806

Published online: 28 May 2014

AbstractforRare disease landscape: will the blockbuster model be replaced? | Full Text | References | PDF (773.9 KB)

3348 Views
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Review Article

Fueling the development of products for rare diseases: the impact of the FDA’s orphan products grants program

Menfo A Imoisili MD MPH, Christine Mueller DO & Katherine I Miller Needleman PhD

Pages: 807-815

Published online: 26 May 2014

AbstractforFueling the development of products for rare diseases: the impact of the FDA’s orphan products grants program | Full Text | References | PDF (528.7 KB)

131 Views
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Review Article

New and emerging treatments of Guillain–Barré syndrome

Helmar C Lehmann, Gang Zhang & Kazim A Sheikh

Pages: 817-829

Published online: 04 Jun 2014

AbstractforNew and emerging treatments of Guillain–Barré syndrome | Full Text | References | PDF (424.8 KB)

167 Views
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Review Article

An overview of current and future treatment options for adults anaplastic oligodendroglial tumors

François Ducray, Caroline Dehais, Jean-Yves Delattre, POLA Network & POLA Network

Pages: 831-840

Published online: 14 Jun 2014

AbstractforAn overview of current and future treatment options for adults anaplastic oligodendroglial tumors | Full Text | References | PDF (481.3 KB)

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Drug Evaluations

Givinostat for the treatment of polycythemia vera

Lisa Pieri, Paola Guglielmelli, Guido Finazzi & Alessandro M Vannucchi

Pages: 841-850

Published online: 23 Jul 2014

Abstractfor Givinostat for the treatment of polycythemia vera | Full Text | References | PDF (311.8 KB)

60 Views
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Cinacalcet hydrochloride for the treatment of hyperparathyroidism

Menaka Sarav & Stuart M Sprague

Pages: 851-863

Published online: 23 Jul 2014

Abstractfor Cinacalcet hydrochloride for the treatment of hyperparathyroidism | Full Text | References | PDF (556.1 KB)

91 Views
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Expert Opinion on Orphan Drugs (Vol.2, Iss.8, 2014)

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